The FDA approved talazoparib (Talzenna) plus enzalutamide on Oct 26, 2026 for adults with metastatic androgen pathway modulation-naïve or -sensitive prostate cancer with HRR gene mutations, as detected by an FDA-authorized test. Approval was supported by the phase 3 TALAPRO-3 trial showing a statistically significant radiographic progression-free survival benefit versus placebo plus enzalutamide (HR 0.48; p<0.0001). Overall survival data were immature at the time of rPFS analysis.
Regulatory approval news: The FDA has granted traditional approval for pembrolizumab (Keytruda) or pembrolizumab plus berahyaluronidase alfa-pmph (Keytruda QLEX) for adult and pediatric patients with unresectable or metastatic tumor mutational burden–high (TMB-H) solid tumors (≥13 mut/Mb by an FDA-authorized test) that progressed after prior treatment and have no satisfactory alternatives. The action converts a prior accelerated approval and updates the TMB-H cutoff from ≥10 to ≥13 mut/Mb based on evidence from KEYNOTE-158.
Regulatory approval: On Oct 19, 2026, the FDA granted accelerated approval to dostarlimab (Jemperli; GSK) for adults with Stage II/III locally advanced rectal cancer that is MSI-H or dMMR, as determined by an FDA-authorized test. Approval was supported primarily by the single-arm AZUR-1 study, which reported a 47% sustained clinical complete response at 12 months (ICR) among 116 evaluable patients, and an ICR ORR of 79%. Safety includes immune-mediated toxicities and infusion reactions.
Regulatory approval: On Oct 14, 2026, the FDA approved an expanded indication for nogapendekin alfa inbakicept-pmln (Anktiva) administered intravesically with BCG for adults with BCG-unresponsive non–muscle invasive bladder cancer (NMIBC). The label now includes patients with high-grade papillary-only tumors, revising the prior indication limited to carcinoma in situ (CIS) with or without papillary tumors. The expansion was based on FDA review and extrapolation from previously demonstrated efficacy in CIS-containing disease.
Research news (Oct 8, 2026) from the US National Cancer Institute highlights decades of federally funded work that helped convert RAS—implicated in roughly one-third of cancers and >90% of pancreatic cancers—from an “undruggable” target to one with multiple inhibitors. The piece cites FDA approval of sotorasib (2021) and accelerated approval of adagrasib (2022) for KRAS G12C–mutant NSCLC, and describes newer, broader-acting RAS inhibitors (e.g., daraxonrasib) and additional investigational agents in clinical trials.
Regulatory approval: On Oct 8, 2026, the FDA approved atezolizumab (Tecentriq) plus a fluoropyrimidine and oxaliplatin for adjuvant treatment of adult and pediatric patients ≥2 years with resected Stage III mismatch repair deficient (dMMR) colon cancer. The FDA also approved subcutaneous atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) plus the same chemotherapy for patients ≥12 years and ≥40 kg. Approval was supported by ATOMIC/ML39057 showing improved investigator-assessed DFS (HR 0.50).
Regulatory approval: On Oct 7, 2026, the FDA approved tucatinib (Tukysa) plus trastuzumab and pertuzumab as maintenance therapy for adults with unresectable locally advanced or metastatic HER2-positive breast cancer without progression after induction with trastuzumab/pertuzumab and a taxane. Approval was supported by HER2CLIMB-05 (N=654), showing investigator-assessed PFS improvement (median 24.9 vs 16.3 months; HR 0.64; p<0.0001). OS data were not mature at the time of PFS analysis.
Regulatory approval: On Oct 2, 2026, the FDA approved pirtobrutinib (Jaypirca; Eli Lilly) for adult patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) with no known 17p deletion. Approval was supported by the randomized, open-label BRUIN CLL-313 trial, in which independent review committee–assessed progression-free survival favored pirtobrutinib versus bendamustine plus a rituximab product. Overall survival data were immature at the primary analysis.
Regulatory approval: On Sept 24, 2026, the FDA approved belzutifan (Welireg) plus lenvatinib (Lenvima) for adults with advanced renal cell carcinoma with a clear cell component following prior PD-1/PD-L1 inhibitor therapy. Approval was supported by the open-label, randomized LITESPARK-011 trial (n=747), which showed a statistically significant improvement in PFS versus cabozantinib; final OS analysis was not statistically significant. Recommended dosing is lenvatinib 20 mg plus belzutifan 120 mg once daily.
Regulatory approval: On September 23, 2026, the FDA approved lirafugratinib (Lyrfigtu; Elevar Therapeutics), a kinase inhibitor, for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene fusion or other rearrangement. Approval was supported by the single-arm REFOCUS trial (NCT04526106; n=116), which reported an ORR of 46% (95% CI, 36–55) and a median DOR of 11.8 months (95% CI, 7.5–13.0).
Research news: The US National Cancer Institute (NCI) and Cancer Research UK (CRUK) highlighted their Cancer Grand Challenges initiative, a global, interdisciplinary program co-founded in 2020 to tackle persistent, large-scale barriers in cancer prevention, detection, and treatment. The program supports international teams with time and resources to pursue high-risk ideas, and includes a “Future Leaders” component for early-career researchers. Initial teams were announced in 2022, with additional cohorts in 2024 and 2026.
Regulatory approval: On Sept 18, 2026, the FDA approved imlunestrant (Inluriyo) plus abemaciclib (Verzenio) for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer (FDA-authorized test required) after progression on at least one line of endocrine therapy. The companion diagnostic Guardant360 CDx was also approved. Approval was supported by EMBER-3, where benefit was reported as concentrated in the ESR1-mutated population; OS data were immature at interim analysis.
Research news (Sept 14, 2026): In an NCI Q&A for Childhood Cancer Awareness Month, Brigitte C. Widemann, MD, highlighted recent pediatric oncology advances and infrastructure efforts. She cited an NCI-supported Children’s Oncology Group phase 3 trial in newly diagnosed B‑cell acute lymphoblastic leukemia in which adding blinatumomab to chemotherapy substantially improved disease-free survival by reducing relapses. She also noted FDA approval of atezolizumab for adults and children ≥2 years with advanced alveolar soft part sarcoma, based on an NCI-led ~50-patient trial.
Regulatory approval: On Sept 9, 2026, the FDA granted accelerated approval to sevabertinib (Hyrnuo; Bayer), a kinase inhibitor, for adults with locally advanced or metastatic non-squamous NSCLC whose tumors harbor HER2 (ERBB2) tyrosine kinase domain activating mutations detected by an FDA-authorized test. The decision expands a prior accelerated approval that was limited to patients who had received prior systemic therapy. Efficacy was supported by single-arm SOHO-01 data in previously untreated patients.
Regulatory approval: On Sept 4, 2026, the FDA granted accelerated approval to camizestrant (Etcamah) plus a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib) for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer with an ESR1 mutation detected during aromatase inhibitor (AI) + CDK4/6 inhibitor therapy using an FDA-authorized test. Approval was based on SERENA-6 PFS results; OS was not mature. Guardant360 CDx was approved as a companion diagnostic.
Regulatory approval: On Aug 26, 2026, the FDA approved daraxonrasib (RASONQUE), a RAS GTPase family inhibitor, for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for multiagent systemic therapy. Approval was supported by the phase 3 RASolute 302 trial, which reported statistically significant improvements in OS, PFS, and ORR versus physician’s choice SOC chemotherapy in both the RAS G12 subgroup and the overall population.
Regulatory approval: On Aug 25, 2026, the FDA approved zanidatamab-hrii (Ziihera) with fluoropyrimidine/platinum chemotherapy and tislelizumab-jsgr (Tevimbra) as first-line treatment for adults with HER2-positive (IHC 3+ or IHC 2+/ISH+) unresectable locally advanced or metastatic gastric, gastroesophageal junction, or esophageal adenocarcinoma (FDA-approved test required). Zanidatamab-hrii with chemotherapy was also approved for HER2-positive IHC 3+ disease. Approvals were supported by HERIZON-GEA-01 efficacy and safety results.
Regulatory approval: On Aug 13, 2026, the FDA granted accelerated approval to iberdomide (Zenbexus) with subcutaneous daratumumab and hyaluronidase-fihj plus dexamethasone for adults with multiple myeloma after ≥1 prior line including a proteasome inhibitor and an immunomodulatory agent. Approval was supported by EXCALIBER-RRMM, where MRD-negative complete response rates were 41% with IberDd vs 21% with DVd. Labeling includes boxed warnings for embryo-fetal toxicity and serious thromboembolism; access is via ZENBEXUS REMS.
Research news: In an NCI commentary dated Aug 6, 2026, Anthony Letai, MD, PhD, argues that “functional precision medicine”—testing a patient’s living cancer cells against therapies—could complement genomics-based precision oncology. Letai cites past laboratory sensitivity testing that helped advance venetoclax toward later FDA approval in CLL, while emphasizing that broader clinical utility of functional testing will require rigorous evidence, standardized workflows, and practical implementation pathways.
Regulatory approval: On July 31, 2026, the FDA approved lutetium Lu 177 vipivotide tetraxetan (Pluvicto) in combination with androgen receptor pathway inhibitor (ARPI) therapy for adults with PSMA-positive metastatic androgen pathway modulation-naïve or -sensitive prostate cancer. Approval was supported by the open-label, randomized PSMAddition trial showing a statistically significant rPFS improvement versus ARPI alone (HR 0.72; p=0.002); median rPFS was not reached. OS data were immature at the time of analysis.
Regulatory approval: The FDA granted accelerated approval (Aug 6, 2026) to vusolimogene oderparepvec-wtpg (Tudriqev) plus nivolumab for adult unresectable advanced cutaneous melanoma that progressed on a PD-1–blocking antibody regimen. Approval was based on objective response rate and duration of response from the single-arm IGNYTE trial. Confirmatory post-approval studies are required, and continued approval may depend on verification of clinical benefit.
Regulatory approval: On July 22, 2026, the FDA approved zidesamtinib (Jideytro; Nuvalent) for adults with locally advanced or metastatic ROS1-positive NSCLC after at least one prior ROS1 tyrosine kinase inhibitor. Approval was supported by the single-arm ARROS-1 trial (N=117), with confirmed ORR 44% (95% CI, 34–53) by BICR. Key labeled risks include CNS adverse reactions, QTc prolongation, ILD/pneumonitis, and others.
Research news (July 21, 2026) from an NCI “Your NCI” blog post reviews how decades of NIH/NCI-funded basic and translational science contributed to the development of immune checkpoint inhibitors. The article outlines discovery of immune “brakes” CTLA-4 and PD-1 and how blocking these pathways can restore antitumor T-cell activity. It notes that checkpoint inhibitors such as pembrolizumab (Keytruda) now have more than 40 approved treatment indications across multiple cancers.
Regulatory approval: On July 14, 2026, the FDA granted traditional approval to selpercatinib (Retevmo) for adult and pediatric patients aged ≥2 years with locally advanced or metastatic RET fusion–positive solid tumors (FDA-approved test) that progressed after prior systemic therapy or lack satisfactory alternatives. The decision converts prior accelerated approvals (adults in 2022; pediatrics ≥2 years in 2024). Approval was supported by LIBRETTO-001 and LIBRETTO-121, with reported ORR and DOR in a non-NSCLC/non-thyroid cohort.
Regulatory approval: On July 14, 2026, the FDA approved gedatolisib (Revtorpyk; Celcuity) with fulvestrant, with or without palbociclib, for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer without a detected PIK3CA mutation after progression on/after at least one line of endocrine therapy in the metastatic setting. Approval was supported by VIKTORIA-1 Study 1, where gedatolisib-containing arms significantly improved PFS versus fulvestrant alone; OS data were not mature.
Regulatory approval: On July 10, 2026, the FDA approved pembrolizumab (Keytruda) or pembrolizumab with berahyaluronidase alfa-pmph (Keytruda Qlex), each in combination with enfortumab vedotin-ejfv (Padcev), as perioperative therapy (neoadjuvant before surgery followed by adjuvant after cystectomy) for adults with muscle invasive bladder cancer (MIBC). The approval expands the prior perioperative regimen indication from cisplatin-ineligible patients to all patients with MIBC who are candidates for cystectomy.
On July 9, 2026, the FDA approved isatuximab-irfc (Sarclisa Escena; Sanofi-Aventis U.S. LLC) for subcutaneous injection for multiple myeloma across three combination-regimen indications: with pomalidomide/dexamethasone after ≥1 prior line including lenalidomide and a proteasome inhibitor; with carfilzomib/dexamethasone for relapsed/refractory disease after 1–3 prior lines; and with bortezomib/lenalidomide/dexamethasone for newly diagnosed, transplant-ineligible adults.
Regulatory approval: On June 30, 2026, the FDA approved Tregzi (HSPC + allogeneic regulatory T cells + conventional T cells; Orca Bio) for use with a myeloablative matched-donor HSCT regimen in adults with hematologic malignancies to support hematopoietic/immunologic reconstitution and improve chronic GVHD-free survival. In the open-label randomized Precision-T trial (NCT05316701) in acute leukemias/MDS, cGVHD-free survival favored Tregzi versus unmanipulated allograft with standard prophylaxis (HR 0.26).
Regulatory approval: On June 24, 2026, the FDA approved sacituzumab govitecan-hziy (Trodelvy) for two first-line indications in adults with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC): (1) monotherapy for patients not candidates for PD-1/PD-L1 inhibitor–based therapy (ASCENT-03) and (2) in combination with pembrolizumab (or pembrolizumab and berahyaluronidase alfa-pmph) for PD-L1–positive disease (CPS ≥10) by an FDA-authorized test (ASCENT-04/KEYNOTE D-19).
Regulatory approval: On June 24, 2026, the FDA approved palbociclib (Ibrance) in combination with trastuzumab, with or without pertuzumab, and endocrine therapy for maintenance treatment of adults with HR-positive, HER2-positive locally advanced or metastatic breast cancer following induction therapy. The decision was supported by the randomized, open-label PATINA trial, which showed a statistically significant improvement in investigator-assessed PFS versus trastuzumab-based maintenance plus endocrine therapy alone; OS data were not mature.
Research news from ASCO 2026 highlights progress in RAS-targeted oncology and ongoing efforts to streamline US clinical trial activation. In an NCI Director commentary dated June 16, 2026, Dr. Anthony Letai described a plenary phase III presentation reporting that daraxonrasib, a RAS(on) inhibitor, nearly doubled median overall survival versus chemotherapy in previously treated advanced pancreatic cancer, while noting remaining uncertainties about safety. He also summarized stakeholder discussions, including an FDA-hosted panel, on reducing operational inefficiencies that delay trial activation.
Regulatory approval: On June 12, 2026, the FDA approved belzutifan (Welireg) plus pembrolizumab (Keytruda) or pembrolizumab/berahyaluronidase alfa-pmph (Keytruda Qlex) for adjuvant treatment of adults with renal cell carcinoma with a clear cell component at intermediate-high or high risk of recurrence after nephrectomy, including after nephrectomy and resection of metastatic lesions. Approval was supported by DFS benefit in LITESPARK-022; OS data were not mature at interim analysis.
Regulatory approval: On June 12, 2026, the FDA approved capivasertib (Truqap) plus abiraterone and prednisone for adults with metastatic androgen pathway modulation-naïve or -sensitive prostate cancer that is PTEN-deficient, as detected by an FDA-authorized test. Approval was supported by CAPItello-281, which showed a statistically significant investigator-assessed rPFS improvement versus placebo plus abiraterone (median 33.2 vs 25.7 months; HR 0.81). OS data were immature at the rPFS analysis.
Regulatory approval: On May 28, 2026, the FDA approved durvalumab (Imfinzi) in combination with bacillus Calmette-Guerin (BCG) for adult patients with BCG-naïve, high-risk non–muscle invasive bladder cancer (NMIBC). Approval was supported by POTOMAC (NCT03528694), where durvalumab+BCG improved investigator-assessed disease-free survival versus BCG alone (HR 0.68; 95% CI 0.50–0.93; p=0.0154; median DFS not reached in either arm).
Regulatory approval: On May 27, 2026, the FDA approved pivekimab sunirine-pvzy (Decnupaz; AbbVie), a CD123-directed antibody–alkylating agent conjugate, for adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Approval was supported by the single-arm CADENZA trial (NCT03386513) evaluating CR/CRc in treatment-naïve and relapsed/refractory BPDCN without active CNS disease. The label includes a Boxed Warning for hepatotoxicity, including hepatic veno-occlusive disease.
Regulatory approval: On May 22, 2026, the FDA approved datopotamab deruxtecan-dlnk (Datroway) for adult patients with unresectable or metastatic triple-negative breast cancer who are not candidates for PD-1/PD-L1 inhibitor therapy. Approval was supported by TROPION-Breast02, where datopotamab deruxtecan-dlnk improved PFS (10.8 vs 5.6 months; HR 0.57) and OS (23.7 vs 18.7 months; HR 0.79) versus investigator’s choice chemotherapy. Label includes warnings for ILD/pneumonitis, ocular adverse reactions, stomatitis/oral mucositis, and embryo-fetal toxicity.
Regulatory approval: On May 15, 2026, the FDA approved atezolizumab (Tecentriq) and atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) as adjuvant treatment for adults with muscle-invasive bladder cancer after cystectomy who have circulating tumor DNA molecular residual disease (ctDNA MRD) identified by an FDA-authorized test. Approval was supported by IMvigor011, which showed statistically significant improvements in disease-free and overall survival versus placebo in ctDNA MRD–positive patients.
Regulatory approval: On May 15, 2026, the FDA approved fam-trastuzumab deruxtecan-nxki (T-DXd; Enhertu) for two indications in adults with HER2-positive (IHC 3+ or ISH+) early-stage breast cancer: (1) neoadjuvant T-DXd for 4 cycles followed by taxane/trastuzumab/pertuzumab (THP) for stage II–III disease, and (2) adjuvant T-DXd for patients with residual invasive disease after neoadjuvant trastuzumab (± pertuzumab) plus a taxane. Two HER2 companion diagnostics were also approved.
Regulatory approval news: On May 13, 2026, the FDA granted accelerated approval to sonrotoclax (Beqalzi), a BCL-2 inhibitor, for adults with relapsed or refractory mantle cell lymphoma after at least two prior systemic therapies, including a BTK inhibitor. Approval was supported by the single-arm BGB-11417-201 trial (NCT05471843), where overall response rate was 52% and median duration of response was 15.8 months per independent review.
Regulatory approval: On May 13, 2026, the FDA approved oral decitabine/cedazuridine tablets (Inqovi) in combination with venetoclax for adults with newly diagnosed AML who are ≥75 years or have comorbidities precluding intensive induction chemotherapy. Approval was based on the single-arm, open-label ASTX727-07 trial (n=101), with complete remission (CR) 41.6% (95% CI 31.9–51.8) and median duration of CR not reached. Label warnings include myelosuppression and embryo-fetal toxicity.
On May 8, 2026, the FDA approved zenocutuzumab-zbco (Bizengri) for adults with advanced, unresectable or metastatic cholangiocarcinoma harboring an NRG1 gene fusion with disease progression on or after prior systemic therapy. Approval was supported by the open-label eNRGy trial, in which 19 patients were evaluable for efficacy; confirmed ORR was 36.8% (95% CI 16.3–61.6) and DOR ranged from 2.8 to 12.9 months. Key labeled safety risks include IRR/hypersensitivity/anaphylaxis, ILD/pneumonitis, and left ventricular dysfunction.
Research news: An NIH-funded Cleveland Clinic study reports that androgen loss (including testosterone) accelerated glioblastoma growth in male preclinical models via hypothalamus-pituitary-adrenal (HPA) axis activation, inflammation, and an immunosuppressive brain microenvironment. In an observational SEER analysis of >1,300 men with glioblastoma, supplemental testosterone use for non-cancer reasons was associated with improved survival (38% lower risk of death). Authors note the human findings are non-causal and support prospective clinical trials.
Research news: In a May 4, 2026 Cancer Currents post, NCI Director Anthony Letai summarized scientific themes and program priorities highlighted at AACR 2026 (San Diego, April 19–22). He pointed to ongoing questions in immuno-oncology response variability, growing momentum in cancer therapeutic vaccines (with NCI–FNIH partnership planning multiple trials), advances and standardization needs in functional precision medicine, and the push for shared, interoperable AI tools. NCI also emphasized streamlining early-phase trials, prevention/access, and human-based models.
Regulatory approval: On May 1, 2026, the FDA approved vepdegestrant (Veppanu), a heterobifunctional protein degrader, for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer detected by an FDA-authorized test, after progression following at least one line of endocrine therapy. Approval was supported by VERITAC-2, where blinded independent central review–assessed PFS improved versus fulvestrant in the ESR1-mutated subgroup (median 5.0 vs 2.1 months; HR 0.57).
Research news: NIH-funded investigators report that scSurvival, a machine-learning model designed for single-cell tumor datasets, predicted survival outcomes and highlighted cell populations associated with risk. In testing on clinical single-cell data from >150 patients (including melanoma and liver cancer), the model reportedly outperformed traditional approaches that average signals across tumors or cell types. The study also traced predictions to immune and tumor cell groups, including melanoma populations associated with immunotherapy response. The work is not a regulatory approval.
Research news (March 25, 2026): An NIH-part funded mouse study reported that repeated colitis-like injury can leave a long-lasting, heritable “epigenetic memory” in colonic stem cells, characterized by increased AP-1 transcription factor activity persisting for >100 days after inflammation ended. In tumor-initiation models, mice that had recovered from chronic colitis developed colorectal tumors more rapidly than controls, and pharmacologic/genetic blockade of AP-1 abrogated the pro-tumor effect.
Regulatory approval: On March 25, 2026, the FDA approved relacorilant (Lifyorli), a glucocorticoid receptor antagonist, in combination with nab-paclitaxel for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after 1–3 prior systemic regimens, including at least one with bevacizumab. Approval was supported by the phase 3 ROSELLA trial, which showed improved PFS and OS versus nab-paclitaxel alone. Labeling includes specific contraindications and safety warnings.
Regulatory approval: On March 20, 2026, the FDA granted accelerated approval to nivolumab plus AVD chemotherapy for adult and pediatric patients aged ≥12 years with previously untreated stage III/IV classical Hodgkin lymphoma. Approval was based on SWOG 1826 (CA209-8UT), which showed superior investigator-assessed PFS versus brentuximab vedotin plus AVD (HR 0.42; 95% CI 0.27–0.67; p<0.0001) with median PFS not reached at 13.7 months’ median follow-up.
Regulatory approval: On March 5, 2026, the FDA granted accelerated approval to teclistamab (Tecvayli) in combination with daratumumab hyaluronidase-fihj for adults with relapsed/refractory multiple myeloma after ≥1 prior line including a proteasome inhibitor and an immunomodulatory agent. The decision was supported by the randomized MajesTEC-3 trial, showing a PFS benefit versus investigator’s choice control. The FDA also converted teclistamab monotherapy from accelerated to traditional approval for heavily pretreated disease.
Regulatory approval: On Feb 26, 2026, the FDA granted accelerated approval to zongertinib (Hernexeos) for adults with unresectable or metastatic non-squamous NSCLC whose tumors harbor HER2 (ERBB2) tyrosine kinase domain activating mutations, detected by an FDA-authorized test. Approval was supported by the single-arm Beamion LUNG-1 trial (n=72), reporting ORR 76% (95% CI 65–85) with durability benchmarks at 6 and 12 months.
Regulatory approval: On Feb 24, 2026, the FDA granted traditional approval to encorafenib (Braftovi) in combination with cetuximab and fluorouracil-based chemotherapy for adults with metastatic colorectal cancer harboring a BRAF V600E mutation detected by an FDA-authorized test. The decision was supported by BREAKWATER, which showed improved PFS, OS, and ORR versus chemotherapy-based control regimens in treatment-naïve patients. Encorafenib previously received accelerated approval in 2024 in combination with cetuximab and mFOLFOX6.
Regulatory approval: On Feb 19, 2026, the FDA approved acalabrutinib (Calquence) tablets/capsules in combination with venetoclax (Venclexta) for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Approval was supported by AMPLIFY, which enrolled previously untreated CLL patients without del(17p) or TP53 mutation and showed improved PFS versus investigator’s choice chemotherapy (FCR/BR; HR 0.65; p=0.0038). Labeling includes infection, hemorrhage, cytopenia, and TLS-related warnings.
Regulatory approval: On Feb 10, 2026, the FDA approved pembrolizumab (Keytruda) and pembrolizumab/berahyaluronidase alfa-pmph (Keytruda Qlex) with paclitaxel, with or without bevacizumab, for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal carcinoma whose tumors are PD-L1–positive (CPS≥1) by an FDA-authorized test and who received 1–2 prior systemic regimens. Approval was supported by KEYNOTE-B96, showing improved PFS and OS in PD-L1 CPS≥1 tumors versus placebo combination therapy.
Regulatory approval: On Feb 5, 2026, the FDA approved safety labeling revisions for capecitabine (Xeloda) and fluorouracil (5-FU) to address risks associated with dihydropyrimidine dehydrogenase (DPD) deficiency. Updates emphasize that patients with complete DPD deficiency (due to certain homozygous or compound heterozygous DPYD variants) are at increased risk of early-onset severe, including fatal, toxicity. Labeling now highlights DPYD testing prior to initiation unless immediate treatment is necessary, and provides guidance to avoid use in complete DPD deficiency and individualize dosing in partial deficiency.
Research news: NIH-supported investigators report a plasma biomarker panel that may improve detection of pancreatic ductal adenocarcinoma (PDAC), including early-stage disease. In a retrospective analysis of banked samples, two novel proteins—aminopeptidase N (ANPEP) and polymeric immunoglobulin receptor (PIGR)—were elevated in early-stage PDAC. A four-marker panel (ANPEP, PIGR, CA19-9, THBS2) distinguished cases from non-cases with 91.9% accuracy at a 5% false-positive rate, and identified 87.5% of stage I/II cases. Further prospective testing is needed.
Regulatory approval: On January 27, 2026, the FDA approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for adults with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). Approval was based on the open-label randomized CEPHEUS trial, which showed higher MRD negativity and improved PFS versus VRd alone. Effectiveness has not been established in patients who refused ASCT as initial therapy.
Regulatory approval: On Dec 17, 2025, the FDA approved rucaparib (Rubraca) for adults with deleterious germline and/or somatic BRCA mutation–associated metastatic castration-resistant prostate cancer (mCRPC) previously treated with an androgen receptor–directed therapy. Selection requires an FDA-approved companion diagnostic. Approval was supported by the confirmatory TRITON3 trial showing improved radiographic progression-free survival versus physician’s choice in BRCA-mutant disease; overall survival was not significantly different.
Regulatory approval: On Dec 17, 2025, the FDA approved amivantamab and hyaluronidase-lpuj (Rybrevant Faspro) for subcutaneous injection for adult patients across all indications already approved for IV amivantamab (Rybrevant). The PALOMA-3 trial in EGFR-mutated locally advanced/metastatic NSCLC supported comparability of exposure and showed no notable differences in descriptive ORR/PFS analyses and no evidence of a detrimental OS effect versus IV dosing. Systemic administration reactions were lower with SC dosing.
Regulatory approval: On Dec 15, 2025, the FDA approved fam-trastuzumab deruxtecan-nxki (Enhertu) plus pertuzumab for first-line treatment of adults with unresectable or metastatic HER2-positive (IHC 3+ or ISH+) breast cancer, as determined by an FDA-approved test. Approval was supported by DESTINY-Breast09, where the combination improved BICR-assessed PFS versus taxane/trastuzumab/pertuzumab (THP). The FDA also approved PATHWAY 4B5 IHC and HER2 Dual ISH companion diagnostics.
Regulatory approval: On Dec 12, 2025, the FDA approved niraparib plus abiraterone acetate (Akeega) with prednisone for adults with deleterious or suspected deleterious BRCA2-mutated metastatic castration-sensitive prostate cancer, as determined by an FDA-approved test. Approval was supported by AMPLITUDE (NCT04497844), where radiographic PFS improved in the overall HRR-mutated population, with exploratory analyses suggesting the benefit was primarily driven by the BRCA2-mutated subgroup.
Research news (Dec 10, 2025): An NIH-led whole-genome sequencing analysis of >1,000 lung cancers, including 542 lung adenocarcinomas, linked an aggressive tumor subgroup enriched for the ID2 mutational signature (single base-pair deletion) to reactivation of LINE-1 (L1) mobile DNA elements. The authors suggest L1 reactivation could contribute to rapid tumor evolution. The study also associated KRAS-mutant tumors with rapid clonal evolution and EGFR-mutant tumors with greater subclonal architecture and a more prolonged evolutionary course.
Regulatory approval: On Dec 4, 2025, the FDA approved lisocabtagene maraleucel (Breyanzi) for adults with relapsed or refractory marginal zone lymphoma (MZL) after at least two prior lines of systemic therapy. Approval was supported by the single-arm, open-label TRANSCEND FL-MZL Cohort (NCT04245839). In the intention-to-treat population (n=77 leukapheresed), ORR was 84.4% and CRR 55.8%; median duration of response was not reached.
Regulatory approval news: On Dec 3, 2025, the FDA granted traditional approval to pirtobrutinib (Jaypirca; Eli Lilly) for adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) previously treated with a covalent BTK inhibitor. Approval was supported by the randomized BRUIN-CLL-321 trial, which showed improved independent review committee–assessed progression-free survival versus investigator’s choice regimens. Updated overall survival results were reported with crossover permitted.
Regulatory approval: On Nov 25, 2025, the FDA approved durvalumab (Imfinzi) with perioperative FLOT chemotherapy as neoadjuvant and adjuvant therapy, followed by durvalumab monotherapy, for adults with resectable gastric or gastroesophageal junction adenocarcinoma. Approval was supported by the phase 3 MATTERHORN trial (NCT04592913), which showed improved event-free survival versus placebo + FLOT (HR 0.71; p<0.001), with overall survival data immature (median not reached in both arms; HR 0.78).
Regulatory approval: On Nov 21, 2025, the FDA approved pembrolizumab (Keytruda) or pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex) in combination with enfortumab vedotin-ejfv (Padcev) as neoadjuvant therapy followed by adjuvant therapy after cystectomy for adults with muscle-invasive bladder cancer who are ineligible for cisplatin. Approval was supported by KEYNOTE-905/EV-303, which showed statistically significant improvements in event-free and overall survival versus immediate surgery alone.
Regulatory approval: On Nov 19, 2025, the FDA granted traditional approval to tarlatamab-dlle (Imdelltra; Amgen) for adults with extensive-stage small cell lung cancer (ES-SCLC) whose disease progressed on or after platinum-based chemotherapy. The drug previously received accelerated approval for this indication in 2024. Approval was supported by the randomized DeLLphi-304 trial, which showed improved overall survival versus investigator’s choice chemotherapy, with boxed warnings for cytokine release syndrome and neurologic toxicity (including ICANS).
Regulatory approval: On Nov 19, 2025, the FDA approved selumetinib (KOSELUGO) for adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas. Approval was supported by the randomized, double-blind, placebo-controlled KOMET trial (NCT04924608), in which confirmed ORR by end of Cycle 16 was 20% with selumetinib vs 5% with placebo (p=0.011); 86% of responders had DOR ≥6 months. Label includes multiple safety warnings.
Regulatory approval: On Nov 19, 2025, the FDA granted traditional approval to subcutaneous daratumumab and hyaluronidase-fihj (Darzalex Faspro) with bortezomib/cyclophosphamide/dexamethasone (VCd) for newly diagnosed light chain (AL) amyloidosis. The indication previously received accelerated approval in 2021. Approval was supported by ANDROMEDA, showing improved MOD-PFS (HR 0.47) and overall survival (HR 0.62) versus VCd alone after 61.4 months’ median follow-up.
Regulatory approval news: On Nov 19, 2025, the FDA granted accelerated approval to sevabertinib (Hyrnuo; Bayer), a kinase inhibitor, for adults with locally advanced or metastatic non-squamous NSCLC harboring HER2 (ERBB2) tyrosine kinase domain activating mutations detected by an FDA-approved test, after prior systemic therapy. Approval was supported by the single-arm SOHO-01 trial with confirmed ORR and DOR as key endpoints. FDA also approved the Oncomine Dx Target Test as a companion diagnostic.
Regulatory approval news: On Nov 18, 2025, the FDA approved epcoritamab-bysp (Epkinly; Genmab) with lenalidomide and rituximab for relapsed or refractory follicular lymphoma (FL). The FDA also granted traditional approval to epcoritamab-bysp monotherapy for relapsed or refractory FL after ≥2 prior systemic therapy lines; this monotherapy indication previously received accelerated approval in 2024. The combination approval was based on EPCORE FL-1, which showed superior PFS and ORR versus R2 alone.
The FDA approved ziftomenib (Komzifti), a menin inhibitor, on Nov 13, 2025, for adults with relapsed or refractory AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. The decision was supported by the open-label, single-arm KO-MEN-001 trial (N=112). The primary efficacy basis included CR+CRh rate (21.4%; median duration 5 months) and transfusion independence outcomes. Label warnings include differentiation syndrome, QTc prolongation, and embryo-fetal toxicity.
Regulatory approval: On Nov 13, 2025, the FDA approved Poherdy (pertuzumab-dpzb; Shanghai Henlius Biologics) as an interchangeable biosimilar to Perjeta (pertuzumab). This is the first FDA-approved biosimilar for Perjeta. Poherdy is indicated with trastuzumab plus docetaxel for certain adults with HER2-positive metastatic breast cancer, and with trastuzumab plus chemotherapy in neoadjuvant and adjuvant settings for specified HER2-positive early/locally advanced breast cancer. Labeling includes boxed warnings for left ventricular dysfunction and embryo-fetal toxicity.
Regulatory approval: On Nov 6, 2025, the FDA approved subcutaneous daratumumab and hyaluronidase-fihj (Darzalex Faspro) for adults with high-risk smoldering multiple myeloma (SMM). Approval was supported by AQUILA, an open-label randomized trial (N=390) comparing monotherapy vs active monitoring. The primary endpoint, IRC-assessed PFS (progression to IMWG-defined multiple myeloma or death), favored daratumumab/hyaluronidase (HR 0.49; 95% CI 0.36–0.67; p<0.0001).
Regulatory approval: On Oct 24, 2025, the FDA approved revumenib (Revuforj; Syndax), a menin inhibitor, for adults and pediatric patients ≥1 year with relapsed or refractory AML harboring a susceptible NPM1 mutation who have no satisfactory alternative treatment options. Approval was supported by a single-arm cohort of the open-label, multicenter AUGMENT-101 trial (NCT04065399), with a CR+CRh rate of 23.1% and median CR+CRh duration of 4.5 months.
The FDA on Oct 23, 2025 approved belantamab mafodotin-blmf (Blenrep; GSK) in combination with bortezomib and dexamethasone for adults with relapsed or refractory multiple myeloma after at least two prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent. Approval was supported by DREAMM-7, where BVd improved PFS and OS versus DVd. The label includes a Boxed Warning for ocular toxicity and requires a REMS (BLENREP REMS).
On Oct 8, 2025, the FDA approved cemiplimab-rwlc (Libtayo) for adjuvant treatment of adults with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence after surgery and radiation. Approval was supported by the phase 3 C-POST trial (NCT03969004), which showed improved disease-free survival versus placebo (HR 0.32; p<0.0001). Labeling includes warnings for immune-mediated adverse reactions, infusion-related reactions, HSCT complications, and embryo-fetal toxicity.
Regulatory approval: On Oct 2, 2025, the FDA approved lurbinectedin (Zepzelca) with atezolizumab (Tecentriq) or atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) as maintenance for adults with extensive-stage small cell lung cancer whose disease has not progressed after first-line induction with atezolizumab (or Tecentriq Hybreza), carboplatin, and etoposide. Approval was supported by IMforte (NCT05091567), showing improved OS and PFS versus atezolizumab alone.
Research news: On Sept. 30, 2025, the U.S. Department of Health and Human Services (HHS) announced it will double funding for the NIH Childhood Cancer Data Initiative (CCDI) from $50 million to $100 million. HHS said the increase is intended to accelerate development of improved diagnostics, treatments, and prevention strategies, including through private-sector partnerships applying artificial intelligence. The announcement accompanied a new executive order, “Unlocking Cures for Pediatric Cancer with Artificial Intelligence.”
Research news: On Sept. 29, 2025, HHS Secretary Robert F. Kennedy, Jr. swore in Anthony Letai, MD, PhD, as Director of the US National Cancer Institute (NCI), part of NIH. Letai previously served as a Harvard Medical School professor and Dana-Farber medical oncologist, with decades of work on cancer cell death, treatment development, and predictive biomarkers. NIH Director Jay Bhattacharya cited Letai’s expertise as key to advancing NCI’s mission.
Regulatory approval: On Sept 25, 2025, the FDA approved imlunestrant (Inluriyo; Eli Lilly) for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer after progression on at least one line of endocrine therapy. Approval was supported by EMBER-3, where investigator-assessed median PFS in the ESR1-mutated subgroup was 5.5 vs 3.8 months versus investigator’s choice endocrine therapy (HR 0.62; p=0.0008). The FDA also approved Guardant360 CDx as a companion diagnostic.
Regulatory approval: On Sept 19, 2025, the FDA approved pembrolizumab and berahyaluronidase alfa‑pmph (Keytruda Qlex, Merck) for subcutaneous injection for adult and pediatric patients (≥12 years) across solid-tumor indications already approved for IV pembrolizumab; clinicians are directed to the prescribing information for indication specifics. Approval was supported by PK comparability data and descriptive efficacy outcomes from MK‑3475A‑D77 in treatment‑naïve metastatic NSCLC without EGFR/ALK/ROS1 aberrations.
Regulatory approval: On Sept 10, 2025, the FDA approved selumetinib (KOSELUGO; AstraZeneca) granules and capsules for pediatric patients aged ≥1 year with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. Selumetinib capsules were previously approved for patients aged ≥2 years. The expanded age indication was supported by bridging data between formulations (relative bioavailability in healthy adults) and exposure matching across pediatric studies, with no new safety signals reported.
Regulatory approval: On Sept 9, 2025, the FDA approved gemcitabine intravesical system (Inlexzo; Janssen Biotech) for adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (with or without papillary tumors). Approval was supported by Cohort 2 of the single-arm SunRISe-1 trial (NCT04640623; n=83), which reported an 82% complete response rate (95% CI, 72–90); 51% of responders had duration of response ≥12 months.
Regulatory approval: On Aug 8, 2025, the FDA granted accelerated approval to zongertinib (Hernexeos) for adults with unresectable or metastatic non-squamous NSCLC harboring HER2 (ERBB2) TKD activating mutations (FDA-approved test) after prior systemic therapy. Approval was supported by Beamion LUNG-1 (NCT04886804), where ORR was 75% in patients previously treated with platinum chemotherapy (no prior HER2 TKI/ADC) and 44% in those previously treated with platinum plus a HER2-targeted ADC. The FDA also approved the Oncomine Dx Target Test as a companion diagnostic.
Regulatory approval: On Aug 6, 2025, the FDA granted accelerated approval to dordaviprone (Modeyso; Jazz Pharmaceuticals), a protease activator, for adult and pediatric patients ≥1 year with H3 K27M–mutant diffuse midline glioma and progressive disease following prior therapy. Approval was supported by an integrated analysis of 50 patients from five open-label, non-randomized U.S. trials. ORR by BICR (RANO 2.0) was 22% with median DOR 10.3 months.
Research news: An NIH/NCI-led whole-genome study (Sherlock-Lung) of 871 lung tumors from never-smokers across 28 global locations found fine-particulate air pollution exposure was strongly associated with increased cancer-driving and cancer-promoting genomic changes, including TP53 alterations and tobacco-smoking–associated mutational signatures. Air pollution exposure was also linked to shorter telomeres, suggesting premature telomere shortening. Secondhand smoke showed slightly higher mutation burden and shorter telomeres but no increase in driver mutations or mutational signatures.
Regulatory approval: On July 2, 2025, the FDA granted accelerated approval to linvoseltamab-gcpt (Lynozyfic), a BCMA-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma after at least four prior lines including a PI, an IMiD, and an anti-CD38 monoclonal antibody. Approval was supported by ORR and DOR estimates from the open-label LINKER-MM1 trial. The label includes a Boxed Warning for CRS and neurologic toxicity (including ICANS) and requires a REMS.
Regulatory approval: On July 2, 2025, the FDA granted accelerated approval to sunvozertinib (Zegfrovy) for adults with locally advanced or metastatic NSCLC harboring EGFR exon 20 insertion mutations (FDA-approved test) after progression on/after platinum-based chemotherapy. Approval was supported by WU-KONG1B (NCT03974022), where sunvozertinib 200 mg daily achieved a confirmed ORR of 46% and median DOR of 11.1 months (BIRC). FDA also approved the Oncomine Dx Express Test as a companion diagnostic.
Regulatory approval: On June 23, 2025, the FDA granted accelerated approval to datopotamab deruxtecan-dlnk (Datroway) for adults with locally advanced or metastatic EGFR-mutated NSCLC after prior EGFR-directed therapy and platinum-based chemotherapy. Approval was supported by a pooled subgroup (n=114) from TROPION-Lung05 and TROPION-Lung01, with confirmed ORR 45% (95% CI 35–54) and median DOR 6.5 months (95% CI 4.2–8.4). Key warnings include ILD/pneumonitis and ocular AEs.
Regulatory approval: On June 18, 2025, the FDA approved tafasitamab-cxix (Monjuvi; Incyte) in combination with lenalidomide and rituximab for adults with relapsed or refractory follicular lymphoma. Approval was supported by the phase 3 inMIND trial (NCT04680052), which showed a statistically significant improvement in investigator-assessed progression-free survival versus placebo plus lenalidomide and rituximab (HR 0.43; p<0.0001).
Regulatory approval: On June 12, 2025, the FDA approved mitomycin intravesical solution (Zusduri; UroGen Pharma) for adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. Approval was supported by the single-arm, multicenter ENVISION trial (NCT05243550), in which 78% of 223 evaluable patients achieved a complete response at 3 months; duration of response ranged from 0 to 25+ months, and 79% of responders remained in response for at least 12 months.
Regulatory approval: On June 12, 2025, the FDA approved pembrolizumab (Keytruda) for adults with resectable locally advanced head and neck squamous cell carcinoma whose tumors express PD-L1 (CPS ≥1) by an FDA-approved test. The perioperative regimen includes neoadjuvant pembrolizumab, continuation postoperatively with adjuvant radiotherapy (with or without cisplatin), then pembrolizumab monotherapy. Approval was supported by KEYNOTE-689, which showed longer event-free survival versus standard postoperative RT ± cisplatin in the CPS ≥1 population.
Regulatory approval: On June 11, 2025, the FDA approved taletrectinib (Ibtrozi; Nuvation Bio) for adults with locally advanced or metastatic ROS1-positive non-small cell lung cancer. Approval was based on two multicenter, single-arm, open-label trials (TRUST-I and TRUST-II) assessing confirmed ORR and DOR by blinded independent central review (RECIST v1.1) in ROS1 TKI–naïve and ROS1 TKI–pretreated patients. Labeling includes multiple warnings, and the recommended dose is 600 mg orally once daily on an empty stomach.
Regulatory approval: On June 3, 2025, the FDA approved darolutamide (Nubeqa) for metastatic castration-sensitive prostate cancer (mCSPC). Approval was based on the phase 3 ARANOTE trial (n=669), where darolutamide plus ongoing androgen deprivation significantly improved radiographic progression-free survival vs placebo (HR 0.54; p<0.0001); median rPFS was not reached vs 25 months. Overall survival was not statistically significantly improved at final analysis (HR 0.78).
Research news: NIH investigators reported blood- and urine-based poly-metabolite scores that aim to objectively estimate the percentage of dietary energy from ultra-processed foods. Using observational data from 718 older U.S. adults plus a small randomized crossover feeding trial (n=20), the scores differentiated high (80% energy) vs none (0%) ultra-processed diet phases. Authors note the need for replication in other populations and further study of associations with outcomes including cancer.
Regulatory approval: On May 15, 2025, the FDA approved retifanlimab-dlwr (Zynyz) with carboplatin and paclitaxel for first-line treatment of adults with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). The FDA also approved retifanlimab-dlwr monotherapy for adults with locally recurrent or metastatic SCAC whose disease progressed on or who are intolerant to platinum-based chemotherapy. Approvals were supported by POD1UM-303/InterAACT 2 and POD1UM-202.
Research news (May 14, 2025): NIH investigators report that “stromal disruption”—architectural and cellular changes in breast connective (stromal) tissue detected by machine learning—was associated with higher risk of developing aggressive breast cancer among women with benign breast disease and with poorer survival among women with invasive breast cancer, particularly ER-positive disease. The authors suggest stromal disruption could become an inexpensive tissue biomarker, but additional studies are needed to validate clinical utility and prevention/treatment implications.
Research news (May 14, 2025) highlights rising incidence of several cancers in people under 50, including colorectal, breast, uterine, and kidney cancers, with the sharpest increases reported in ages 20–29. Investigators emphasize that causes remain uncertain and likely multifactorial. An NCI-led US analysis found 14 cancer types increased in at least one <50 age group during 2010–2019, and nine of those also rose in at least one >50 age band, suggesting shared drivers across ages.
Regulatory approval: On May 14, 2025, the FDA granted accelerated approval to telisotuzumab vedotin-tllv (Emrelis) for adults with locally advanced or metastatic, non-squamous NSCLC with high c-Met protein overexpression (≥50% of tumor cells with strong [3+] staining) by an FDA-approved test, after prior systemic therapy. Approval was supported by LUMINOSITY (NCT03539536), with confirmed ORR 35% and median DOR 7.2 months. FDA also approved the VENTANA MET (SP44) RxDx Assay as a companion diagnostic.
Regulatory approval: On May 14, 2025, the FDA approved belzutifan (Welireg; Merck) for adult and pediatric patients aged ≥12 years with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). Efficacy was supported by the open-label LITESPARK-015 trial (cohort A1; n=72), with an objective response rate of 26% (95% CI, 17–38) by blinded independent central review and median duration of response of 20.4 months (95% CI, 8.3–NR).
Research news: An NCI-supported study reports an “Ultra-Rapid” droplet digital PCR (ddPCR) workflow that measured IDH1 R132H and BRAF V600E mutations in intraoperative brain tissue samples in about 15 minutes. In >75 samples from 22 patients, the method’s performance matched standard ddPCR, while also detecting very small numbers of tumor cells (reported as low as five cells/mm²). Authors say the approach is investigational and has not yet been used to actively guide surgery; further refinement and clinical trials are planned.
Regulatory approval: On May 8, 2025, the FDA granted accelerated approval to the co-pack of avutometinib plus defactinib (Avmapki Fakzynja Co-pack; Verastem, Inc.) for adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer who have received prior systemic therapy. Approval was supported by RAMP-201 (NCT04625270), where confirmed ORR was 44% (95% CI 31–58) by blinded independent review; duration of response ranged from 3.3 to 31.1 months.
Research news: UC San Diego investigators report a microfluidic device that measures tumor-cell “stickiness” (adhesion) from surgically removed samples as a potential predictor of metastatic potential. In mouse experiments, less-adherent cells produced more lung metastases than highly adherent cells. In human breast tissue samples from 16 women, normal tissue showed high adhesion, while invasive breast tumor samples were less adherent; DCIS samples showed variable adhesion. Authors emphasize larger studies are needed, including planned studies in DCIS and exploration in lung and prostate cancers.
Regulatory approval news: On May 2, 2025, the FDA approved nivolumab plus ipilimumab for first-line treatment of advanced colorectal cancer with MSI-H or dMMR tumors (~5% of advanced cases). The decision was based on updated CheckMate-8HW results showing longer progression-free survival (PFS) versus nivolumab alone (3-year PFS ~68% vs 51%). Serious adverse events and discontinuations were higher with the combination; treatment-related deaths occurred in both arms.
Regulatory approval: On April 23, 2025, the FDA approved penpulimab-kcqx for adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC). Approvals include first-line use with cisplatin or carboplatin plus gemcitabine, and single-agent use for metastatic non-keratinizing NPC after progression on or after platinum-based chemotherapy and at least one additional prior line. In AK105-304, penpulimab combination improved PFS vs placebo; OS was immature with no detrimental trend reported.
Regulatory approval: On April 11, 2025, the FDA approved nivolumab plus ipilimumab for first-line treatment of adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). Approval was supported by the phase 3 CHECKMATE-9DW trial, which reported median overall survival of 23.7 months with nivolumab/ipilimumab vs 20.6 months with lenvatinib or sorafenib (HR 0.79; 95% CI 0.65–0.96; p<0.0180) and ORR 36.1% vs 13.2% (p<0.0001).
Regulatory approval: On April 8, 2025, the FDA granted accelerated approval to nivolumab plus ipilimumab for adult and pediatric patients ≥12 years with unresectable or metastatic MSI-H or dMMR colorectal cancer. The FDA also converted prior accelerated approval to regular approval for single-agent nivolumab in adult and pediatric patients ≥12 years with MSI-H/dMMR metastatic CRC that progressed after fluoropyrimidine, oxaliplatin, and irinotecan. The approvals were supported by CHECKMATE-8HW PFS and response data; OS comparisons were not yet available at interim analysis.
Regulatory approval: On March 28, 2025, the FDA approved durvalumab (Imfinzi) in combination with gemcitabine and cisplatin as neoadjuvant therapy, followed by single-agent durvalumab as adjuvant therapy after radical cystectomy, for adults with muscle invasive bladder cancer. Approval was supported by the phase III NIAGARA trial (NCT03732677), which showed statistically significant improvements in event-free survival and overall survival versus neoadjuvant chemotherapy followed by surgery alone.
Regulatory approval: On March 28, 2025, the FDA expanded the indication for lutetium Lu 177 vipivotide tetraxetan (Pluvicto) to adults with PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) previously treated with an androgen receptor pathway inhibitor (ARPI) and considered appropriate to delay taxane-based chemotherapy. The decision was supported by PSMAfore (NCT04689828), where Pluvicto improved radiographic progression-free survival versus a change in ARPI; overall survival was not statistically significant, with substantial crossover from the control arm.
Regulatory approval: On March 26, 2025, the FDA approved cabozantinib (Cabometyx) for adult and pediatric patients aged ≥12 years with previously treated, unresectable, locally advanced or metastatic, well-differentiated pancreatic NET (pNET) and well-differentiated extra-pancreatic NET (epNET). Approval was supported by the randomized, double-blind CABINET trial, which showed improved BIRC-assessed PFS vs placebo in both pNET and epNET cohorts; OS data were not mature and crossover may affect OS interpretation.
Regulatory approval: On March 19, 2025, the FDA granted traditional approval to pembrolizumab with trastuzumab plus fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of adults with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma with PD-L1 expression (CPS ≥1). The decision was supported by KEYNOTE-811, which showed statistically significant improvements in overall survival and progression-free survival versus placebo with trastuzumab and chemotherapy. Pembrolizumab had received accelerated approval for this indication in 2021.
Regulatory approval: On Feb 14, 2025, the FDA approved vimseltinib (Romvimza; Deciphera), a kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor (TGCT) when surgical resection would potentially worsen functional limitation or cause severe morbidity. Approval was supported by the randomized, placebo-controlled MOTION trial (NCT05059262), where week-25 blinded ORR was 40% with vimseltinib vs 0% with placebo (p<0.0001). Median DOR was not reached at the time of analysis.
Regulatory approval: On Feb 12, 2025, the FDA approved brentuximab vedotin (Adcetris) with lenalidomide and a rituximab product for adult relapsed/refractory large B-cell lymphoma after ≥2 prior systemic lines, in patients ineligible for autologous HSCT or CAR T-cell therapy. Approval was based on the phase 3 ECHELON-3 trial, which showed improved overall survival versus placebo plus lenalidomide/rituximab (median OS 13.8 vs 8.5 months; HR 0.63).
Regulatory approval: On Feb 11, 2025, the FDA approved mirdametinib (Gomekli; SpringWorks Therapeutics), a kinase inhibitor, for adults and pediatric patients aged ≥2 years with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. Approval was supported by the single-arm ReNeu trial (NCT03962543), which reported confirmed ORR of 41% in adults and 52% in pediatric patients by blinded central review using volumetric MRI.
Regulatory approval: The FDA approved treosulfan (Grafapex; medac GmbH) with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients aged ≥1 year with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Approval was supported by the randomized MC-FludT.14/L Trial II comparing treosulfan vs busulfan with fludarabine, with overall survival as the major efficacy endpoint.
Regulatory approval: On Jan 27, 2025, the FDA approved fam-trastuzumab deruxtecan-nxki (Enhertu) for unresectable or metastatic HR-positive, HER2-low (IHC 1+ or IHC 2+/ISH−) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, as determined by an FDA-approved test, after progression on ≥1 endocrine therapy in the metastatic setting. Approval was supported by DESTINY-Breast06, which showed improved PFS versus physician’s choice chemotherapy; OS data were immature.
Regulatory safety update (FDA, Jan 24, 2025): the FDA issued a communication to raise awareness of updated labeling for capecitabine (Xeloda) and fluorouracil (5‑FU) regarding risks from dihydropyrimidine dehydrogenase (DPD) deficiency. The agency highlights discussing DPD deficiency and testing options with patients before starting therapy, and notes labeling statements that these fluoropyrimidines are not recommended in patients with complete DPD deficiency and that data are insufficient to recommend a specific dose in partial DPD deficiency.
Regulatory approval: On Jan 17, 2025, the FDA approved datopotamab deruxtecan-dlnk (Datroway), a Trop-2–directed antibody–topoisomerase inhibitor conjugate, for adults with unresectable or metastatic HR-positive, HER2-negative breast cancer (IHC 0/1+ or IHC2+/ISH−) previously treated with endocrine-based therapy and chemotherapy for unresectable/metastatic disease. Approval was supported by TROPION-Breast01, which showed improved PFS vs investigator’s choice chemotherapy, while OS was not statistically significant.
Regulatory approval: On Jan 16, 2025, the FDA granted traditional approval for acalabrutinib (Calquence) with bendamustine and rituximab for adults with previously untreated mantle cell lymphoma who are ineligible for autologous HSCT. Approval was supported by the phase 3 ECHO trial (NCT02972840), showing longer PFS vs placebo plus BR (HR 0.73; p=0.016). The FDA also granted traditional approval to single-agent acalabrutinib for previously treated MCL, converting its 2017 accelerated approval.
Regulatory approval: On Jan 16, 2025, the FDA approved sotorasib (Lumakras) with panitumumab (Vectibix) for adult patients with KRAS G12C-mutated metastatic colorectal cancer, as determined by an FDA-approved test, after prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. Approval was supported by CodeBreaK 300, where sotorasib 960 mg plus panitumumab improved PFS versus investigator’s choice SOC (trifluridine/tipiracil or regorafenib). A QIAGEN therascreen companion diagnostic was also approved.
Regulatory approval: On Dec 27, 2024, the FDA approved nivolumab and hyaluronidase-nvhy (Opdivo Qvantig) for subcutaneous injection across approved adult solid-tumor nivolumab indications, as monotherapy, monotherapy maintenance after Opdivo plus Yervoy, or in combination with chemotherapy or cabozantinib. The approval was supported by CHECKMATE-67T in advanced/metastatic clear cell RCC, showing pharmacokinetic comparability to IV nivolumab and ORR 24% vs 18%.
Regulatory approval: On Dec 20, 2024, the FDA granted accelerated approval to encorafenib with cetuximab and mFOLFOX6 for patients with metastatic colorectal cancer harboring a BRAF V600E mutation, as detected by an FDA-approved test. The decision was based on confirmed objective response rate and duration of response from the ongoing, active-controlled BREAKWATER trial. Progression-free survival and overall survival analyses are planned as post-marketing confirmatory evidence.
Regulatory approval: On Dec 18, 2024, the FDA approved Ryoncil (remestemcel-L-rknd), an allogeneic bone marrow–derived mesenchymal stromal cell therapy, for steroid-refractory acute graft-versus-host disease (aGVHD) in pediatric patients aged ≥2 months. Efficacy was assessed in a single-arm study (MSB-GVHD001; n=54) with Grade B–D steroid-refractory aGVHD (excluding Grade B skin alone), reporting a Day 28 overall response rate of 70% and a median response duration of 54 days.
Regulatory approval: On Dec 18, 2024, the FDA approved ensartinib (Ensacove) for adult patients with ALK-positive locally advanced or metastatic NSCLC who have not previously received an ALK inhibitor. Approval was supported by the phase 3 eXALT3 trial comparing ensartinib vs crizotinib, showing statistically significant improvement in blinded independent central review–assessed PFS (HR 0.56). No statistically significant overall survival difference was reported.
Regulatory approval: On Dec 18, 2024, the FDA approved remestemcel-L-rknd (Ryoncil; Mesoblast), an allogeneic bone marrow–derived mesenchymal stromal cell (MSC) therapy, for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged ≥2 months. Approval was supported by the single-arm MSB-GVHD001 study (NCT02336230) in 54 pediatric post-HSCT patients, where Day-28 overall response rate was 70% (95% CI, 56.4–82.0).
Regulatory approval: On Dec 13, 2024, the FDA approved cosibelimab-ipdl (Unloxcyt), a PD-L1–blocking antibody, for adults with metastatic cutaneous squamous cell carcinoma (mCSCC) or locally advanced CSCC (laCSCC) who are not candidates for curative surgery or curative radiation. Approval was supported by Study CK-301-101 (NCT03212404), reporting ORR 47% in mCSCC (n=78) and 48% in laCSCC (n=31); median DOR was not reached in mCSCC and was 17.7 months in laCSCC.
Regulatory approval: On Dec 4, 2024, the FDA granted accelerated approval to zenocutuzumab-zbco (Bizengri) for adults with advanced, unresectable, or metastatic pancreatic adenocarcinoma harboring an NRG1 gene fusion whose disease progressed on or after prior systemic therapy. Efficacy was evaluated in the multicenter, open-label eNRGy trial; in the pancreatic cohort (n=30), confirmed ORR was 40%, with duration of response ranging from 3.7 to 16.6 months. Labeling includes a boxed warning for embryo-fetal toxicity.
Regulatory approval: On Dec 4, 2024, the FDA granted accelerated approval to zenocutuzumab-zbco (Bizengri; Merus N.V.) for adults with advanced, unresectable, or metastatic NRG1 fusion–positive NSCLC or pancreatic adenocarcinoma whose disease progressed on or after prior systemic therapy. Approval was supported by ORR and DOR results from the multicenter, open-label eNRGy trial (NCT02912949). This is described as the first FDA-approved systemic therapy for NRG1 fusion–positive NSCLC or pancreatic adenocarcinoma.
Regulatory approval: On Dec 4, 2024, the FDA approved durvalumab (Imfinzi) for adults with limited-stage small cell lung cancer whose disease has not progressed after concurrent platinum-based chemotherapy and radiation therapy. Approval was supported by the phase 3 ADRIATIC trial, where durvalumab improved overall survival (HR 0.73) and progression-free survival (HR 0.76) versus placebo. Common adverse reactions (≥20%) included pneumonitis/radiation pneumonitis and fatigue.
Regulatory approval news: The FDA granted accelerated approval (Nov 21, 2024) to zanidatamab-hrii (Ziihera), a bispecific HER2-directed antibody, for previously treated unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer, as detected by an FDA-approved test. Approval was supported by the single-arm HERIZON-BTC-01 trial (n=62), with ORR 52% and median DOR 14.9 months. FDA also approved the VENTANA PATHWAY anti-HER-2/neu (4B5) assay as a companion diagnostic.
Regulatory approval: On Nov 19, 2024, the FDA approved updated labeling for fludarabine phosphate (Fludarabine Phosphate Injection, Sandoz) under the Oncology Center of Excellence’s Project Renewal. The label now includes new and revised indications in adult B-cell chronic lymphocytic leukemia (CLL), updated dosing information for use with cyclophosphamide and rituximab, and removal of the boxed warning with related content moved to Warnings and Precautions. Full prescribing information will be posted on Drugs@FDA.
Regulatory approval: On Nov 15, 2024, the FDA approved revumenib (Revuforj; Syndax), a menin inhibitor, for adult and pediatric patients aged ≥1 year with relapsed or refractory acute leukemia with a KMT2A translocation. Approval was supported by a single-arm cohort of the open-label AUGMENT-101 trial (NCT04065399) in 104 patients. The CR+CRh rate was 21.2% with a median duration of 6.4 months (upper CI not estimable).
Regulatory approval: On Nov 8, 2024, the FDA approved obecabtagene autoleucel (Aucatzyl; Autolus), a CD19-directed genetically modified autologous T-cell immunotherapy, for adults with relapsed or refractory CD19-positive B-cell precursor acute lymphoblastic leukemia. Approval was supported by the single-arm FELIX trial (NCT04404660). In 65 evaluable patients, 42% achieved complete remission within 3 months; median duration of that CR was 14.1 months. Boxed warnings include CRS, ICANS, and T-cell malignancies.
Regulatory approval: On Oct 29, 2024, the FDA granted accelerated approval to asciminib (Scemblix; Novartis) for adults with newly diagnosed Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). Approval was supported by ASC4FIRST (NCT04971226), where major molecular response (MMR) at 48 weeks was higher with asciminib than investigator-selected TKIs (68% vs 49%; difference 19%; p<0.001). Safety and dosing details were included in the FDA communication.
Regulatory approval: On Oct 18, 2024, the FDA approved zolbetuximab-clzb (Vyloy), a CLDN18.2-directed cytolytic antibody, in combination with fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are CLDN18.2-positive by an FDA-approved test. Approval was supported by the phase 3 SPOTLIGHT and GLOW trials showing improved PFS and OS versus chemotherapy plus placebo.
Regulatory approval: On Oct 10, 2024, the FDA approved inavolisib (Itovebi) in combination with palbociclib and fulvestrant for adults with endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy. Approval was supported by INAVO120, which showed improved investigator-assessed PFS versus placebo plus palbociclib/fulvestrant; interim OS analysis did not reach statistical significance.
Regulatory approval: On October 3, 2024, the FDA approved nivolumab with platinum-doublet chemotherapy as neoadjuvant therapy, followed by adjuvant single-agent nivolumab after surgery, for adults with resectable (tumors ≥4 cm and/or node-positive) NSCLC without known EGFR mutations or ALK rearrangements. Approval was supported by CHECKMATE-77T, where event-free survival was improved versus chemotherapy (median not reached vs 18.4 months; HR 0.58). Interim OS was descriptive and showed no detriment.
Regulatory approval: On Sept 25, 2024, the FDA approved osimertinib (Tagrisso) for adults with locally advanced, unresectable stage III NSCLC whose disease has not progressed during or after concurrent or sequential platinum-based chemoradiation and whose tumors harbor EGFR exon 19 deletions or exon 21 L858R mutations (FDA-approved test required). Approval was supported by LAURA (NCT03521154), where osimertinib significantly improved PFS vs placebo (HR 0.16; median 39.1 vs 5.6 months). OS data were immature.
Regulatory approval: On September 20, 2024, the FDA approved isatuximab-irfc (Sarclisa) in combination with bortezomib, lenalidomide, and dexamethasone for adults with newly diagnosed multiple myeloma who are not eligible for autologous stem cell transplant. Approval was supported by the phase 3 IMROZ trial, which showed improved independent review committee–assessed PFS versus VRd alone (HR 0.60; p=0.0009); median PFS was not reached with Isa-VRd and was 54.3 months with VRd.
Regulatory approval: On Sept 19, 2024, the FDA approved amivantamab-vmjw (Rybrevant) with carboplatin and pemetrexed for adults with locally advanced or metastatic NSCLC harboring EGFR exon 19 deletions or exon 21 L858R mutations whose disease progressed on or after an EGFR TKI. Approval was supported by the phase 3 MARIPOSA-2 trial, which showed improved BICR-assessed PFS and ORR versus carboplatin/pemetrexed; interim OS was not statistically significant.
Regulatory approval: On Sept 17, 2024, the FDA approved ribociclib (Kisqali) with an aromatase inhibitor, and the Kisqali Femara Co-Pack (ribociclib + letrozole), for adjuvant treatment of adults with HR-positive, HER2-negative stage II–III early breast cancer at high risk of recurrence. Approval was supported by the phase 3 NATALEE trial, which showed improved invasive disease-free survival versus a non-steroidal aromatase inhibitor alone; overall survival was immature at final iDFS analysis.
Regulatory approval: On Sept 17, 2024, the FDA approved pembrolizumab (Keytruda) with pemetrexed and platinum chemotherapy as first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma. The decision was based on KEYNOTE-483, a randomized open-label trial in patients without prior systemic therapy for advanced/metastatic disease, showing a statistically significant overall survival improvement versus chemotherapy alone (median OS 17.3 vs 16.1 months).
Regulatory approval: On Sep 17, 2024, the FDA approved pembrolizumab (Keytruda) with pemetrexed and platinum chemotherapy as first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma. Approval was supported by the randomized open-label KEYNOTE-483 trial, which showed a statistically significant overall survival benefit versus chemotherapy alone (median OS 17.3 vs 16.1 months; HR 0.79; p=0.0162).
Regulatory approval: On Sept 12, 2024, the FDA approved atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) for subcutaneous injection for all adult indications held by IV atezolizumab (Tecentriq), including NSCLC, SCLC, HCC, melanoma, and ASPS. Approval was supported by IMscin001, which met prespecified pharmacokinetic comparability criteria versus IV atezolizumab in previously treated, locally advanced/metastatic NSCLC; descriptive efficacy outcomes appeared similar between formulations.
Regulatory approval: On Aug 19, 2024, the FDA approved lazertinib (Lazcluze) plus amivantamab-vmjw (Rybrevant) for first-line treatment of locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R mutations detected by an FDA-approved test. Approval was supported by MARIPOSA (NCT04487080), where the combination improved BICR-assessed PFS versus osimertinib (HR 0.70; median 23.7 vs 16.6 months). OS data were immature.
Regulatory approval: On Aug 15, 2024, the FDA approved durvalumab (Imfinzi) with platinum-containing chemotherapy as neoadjuvant therapy, followed by adjuvant single-agent durvalumab after surgery, for adults with resectable (tumors ≥4 cm and/or node-positive) NSCLC without known EGFR mutations or ALK rearrangements. Approval was supported by AEGEAN (NCT03800134), which showed improved event-free survival (HR 0.68) and higher pCR (17% vs 4.3%) versus placebo.
Regulatory approval: On Aug 14, 2024, the FDA approved axatilimab-csfr (Niktimvo), a CSF-1R–blocking antibody, for chronic graft-versus-host disease after failure of at least two prior systemic therapy lines in adults and pediatric patients weighing ≥40 kg. Approval was supported by the randomized, open-label AGAVE-201 trial. In 79 patients treated at the recommended dose, ORR through Cycle 7 Day 1 was 75% (95% CI 64–84). Priority review was granted; orphan drug and fast track designations were in place.
Regulatory approval: On Aug 6, 2024, the FDA approved vorasidenib (Voranigo), an IDH1/IDH2 inhibitor, for adult and pediatric patients ≥12 years with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery. Approval was supported by the phase 3 INDIGO trial (NCT04164901), which showed improved PFS (HR 0.39) and time to next intervention (HR 0.26) versus placebo. Common AEs included fatigue and headache; notable lab abnormalities included transaminase elevations.
Regulatory approval: On Aug 2, 2024, the FDA granted accelerated approval to afamitresgene autoleucel (TECELRA), a MAGE-A4–directed genetically modified autologous T-cell immunotherapy, for adults with unresectable or metastatic synovial sarcoma after prior chemotherapy, who are HLA-A*02:01P/-A*02:02P/-A*02:03P/-A*02:06P positive and whose tumors express MAGE-A4 per FDA-approved/cleared companion diagnostics. Approval was supported by the single-arm SPEARHEAD-1 Cohort 1 trial (ORR 43.2%).
Regulatory approval: On Aug 2, 2024, the FDA granted accelerated approval to Tecelra (afamitresgene autoleucel), a MAGE-A4–directed genetically modified autologous T-cell immunotherapy, for certain adults with unresectable or metastatic synovial sarcoma after prior chemotherapy. Eligibility includes specific HLA-A*02 alleles and MAGE-A4–positive tumors identified by FDA-approved/cleared companion diagnostics. In SPEARHEAD-1 Cohort 1 (single-arm), overall response rate was 43.2% with median duration of response 6 months. Label includes a boxed warning for serious or fatal cytokine release syndrome.
Regulatory approval: On Aug 1, 2024, the FDA approved dostarlimab-gxly (Jemperli) with carboplatin/paclitaxel followed by dostarlimab monotherapy for adult patients with primary advanced or recurrent endometrial cancer, expanding beyond the prior dMMR/MSI-H–restricted indication. Approval was supported by the phase 3 RUBY trial (N=494), which showed statistically significant improvements in overall survival and progression-free survival in the overall population versus placebo plus chemotherapy.
Regulatory approval: On July 30, 2024, the FDA approved daratumumab and hyaluronidase-fihj (Darzalex Faspro) in combination with bortezomib, lenalidomide, and dexamethasone for induction and consolidation in patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant (ASCT). The decision was supported by the phase 3 PERSEUS trial, which showed improved progression-free survival vs VRd alone (HR 0.40; p<0.0001), with median PFS not reached in either arm.
Regulatory approval: On June 26, 2024, the FDA granted accelerated approval to subcutaneous epcoritamab-bysp (Epkinly), a CD20-directed CD3 T-cell engager, for adults with relapsed or refractory follicular lymphoma after ≥2 prior systemic therapy lines. Approval was based on the single-arm EPCORE NHL-1 trial (NCT03625037), where ORR was 82% with 60% complete responses; median duration of response was not reached at 14.8 months’ median follow-up among responders. A confirmatory phase 3 trial is ongoing.
Regulatory approval: On June 21, 2024, the FDA granted accelerated approval to adagrasib (Krazati) plus cetuximab for adults with KRAS G12C–mutated locally advanced or metastatic colorectal cancer (as determined by an FDA-approved test) previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. Approval was supported by KRYSTAL-1 (single-arm), where confirmed ORR was 34% with a median DOR of 5.8 months.
Regulatory approval: On June 17, 2024, the FDA approved pembrolizumab plus carboplatin/paclitaxel, followed by single-agent pembrolizumab, for adult patients with primary advanced or recurrent endometrial carcinoma. Approval was supported by KEYNOTE-868/NRG-GY018, which showed improved investigator-assessed PFS versus placebo plus chemotherapy in both dMMR (HR 0.30) and pMMR (HR 0.60) cohorts. Rash occurred more often with pembrolizumab plus chemotherapy.
Regulatory approval: On June 14, 2024, the FDA approved blinatumomab (Blincyto) for adults and pediatric patients ≥1 month old with CD19-positive, Philadelphia chromosome–negative B-cell precursor acute lymphoblastic leukemia in the consolidation phase of multiphase chemotherapy. Approval was supported by randomized trials in adults (E1910) and in pediatric/young adult patients (Study 20120215), showing improved overall survival versus chemotherapy-based comparators. The review used Project Orbis and RTOR and received priority review, breakthrough, and orphan designations.
Regulatory approval: On June 14, 2024, the FDA approved durvalumab with carboplatin plus paclitaxel followed by durvalumab maintenance for adult patients with primary advanced or recurrent mismatch repair–deficient (dMMR) endometrial cancer. The decision was supported by DUO-E (NCT04269200), where an exploratory analysis in 95 patients with dMMR tumors showed median PFS not reached with durvalumab vs 7 months with placebo (HR 0.42; 95% CI, 0.22–0.80).
Regulatory approval news: In an FDA D.I.S.C.O. Burst Edition update, the FDA granted accelerated approvals to repotrectinib (Augtyro) for NTRK gene fusion–positive solid tumors in adults and pediatric patients ≥12 years (June 13, 2024) and to adagrasib (Krazati) plus cetuximab for previously treated KRAS G12C–mutated locally advanced/metastatic colorectal cancer (June 21, 2024). Approvals were supported by single-arm trials (TRIDENT-1 and KRYSTAL-1) reporting overall response rate and duration of response.
Regulatory approval: On June 13, 2024, the FDA granted accelerated approval to repotrectinib (AUGTYRO) for adults and pediatric patients aged ≥12 years with NTRK gene fusion–positive solid tumors that are locally advanced/metastatic or where surgery would likely cause severe morbidity, and that have progressed after treatment or have no satisfactory alternative therapy. Approval was supported by the single-arm TRIDENT-1 trial, reporting confirmed ORR of 58% in TRK TKI–naïve and 50% in TRK TKI–pretreated patients.
Regulatory approval: On June 12, 2024, the FDA granted traditional approval to selpercatinib (Retevmo) for adults and pediatric patients aged ≥2 years with advanced or metastatic RET fusion–positive thyroid cancer requiring systemic therapy and who are radioactive iodine (RAI)-refractory (if RAI is appropriate). Efficacy was primarily supported by LIBRETTO-001, reporting high overall response rates in both previously treated and systemic therapy–naïve cohorts; supportive pediatric/young adult data came from LIBRETTO-121. Common adverse reactions and key grade 3/4 lab abnormalities were reported.
Regulatory approval: On June 6, 2024, the FDA approved imetelstat (Rytelo; Geron) for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) and transfusion-dependent anemia (≥4 RBC units over 8 weeks) who have not responded to, lost response to, or are ineligible for erythropoiesis-stimulating agents. Approval was supported by the IMerge randomized, double-blind, placebo-controlled trial showing higher ≥8-week and ≥24-week RBC transfusion independence rates versus placebo.
Regulatory approval: On May 30, 2024, the FDA approved lisocabtagene maraleucel (Breyanzi) for adult patients with relapsed or refractory mantle cell lymphoma after at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor. Approval was supported by the single-arm TRANSCEND-MCL trial, in which the primary efficacy population (n=68) had an IRC-assessed ORR of 85.3% and CRR of 67.6%. The product carries a REMS for risks of CRS and neurologic toxicities.
Regulatory approval: On May 29, 2024, the FDA granted accelerated approval to selpercatinib (Retevmo) for pediatric patients aged ≥2 years with (1) advanced/metastatic RET-mutant medullary thyroid cancer requiring systemic therapy; (2) advanced/metastatic RET fusion–positive thyroid cancer requiring systemic therapy that is radioactive iodine–refractory (if RAI is appropriate); and (3) locally advanced/metastatic RET fusion–positive solid tumors with progression after prior systemic therapy or no satisfactory alternatives. Approval was supported by LIBRETTO-121 (single-arm), with confirmed ORR 48% and median DoR not reached.
Regulatory approval: On May 29, 2024, the FDA granted accelerated approval to selpercatinib (Retevmo) for pediatric patients ≥2 years with specified RET-altered advanced/metastatic thyroid cancers and RET fusion–positive solid tumors. The decision was supported by the single-arm LIBRETTO-121 trial (NCT03899792), which reported a confirmed ORR of 48% (95% CI, 28–69) in the primary efficacy population, with median DOR not reached. This expands prior accelerated approvals that included adults and patients ≥12 years.
Regulatory approval news: On May 29, 2024, the FDA granted accelerated approval to selpercatinib (Retevmo) for pediatric patients aged ≥2 years with advanced or metastatic medullary thyroid cancer (MTC) harboring a RET mutation detected by an FDA-approved test, who require systemic therapy. The FDA cited LIBRETTO-531, where selpercatinib improved progression-free survival versus cabozantinib or vandetanib in adults/adolescents with RET-mutant MTC. Common adverse reactions included hypertension, edema, dry mouth, fatigue, and diarrhea.
Regulatory approval: On May 16, 2024, the FDA granted accelerated approval to tarlatamab-dlle (Imdelltra; Amgen) for extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. Approval was based on ORR and DOR from the open-label DeLLphi-301 study (n=99). ORR was 40% (95% CI 31–51) with a median DOR of 9.7 months (range 2.7–20.7+). Continued approval may be contingent on confirmatory evidence of clinical benefit.
Regulatory approval: On May 15, 2024, the FDA granted accelerated approval to lisocabtagene maraleucel (Breyanzi) for adults with relapsed or refractory follicular lymphoma after ≥2 prior lines of systemic therapy. Approval was supported by the phase 2, single-arm TRANSCEND-FL trial in a primary efficacy population of 94 PET-positive patients. ORR was 95.7% and median DOR was not reached at median follow-up of 16.8 months. A REMS is required for CRS and neurologic toxicities.
Regulatory approval: On April 29, 2024, the FDA granted traditional approval to tisotumab vedotin-tftv (Tivdak) for recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. The drug previously held accelerated approval for this indication. Approval is supported by innovaTV 301, a randomized trial (n=502) showing improved overall survival vs investigator’s choice chemotherapy (median OS 11.5 vs 9.5 months; HR 0.70; p=0.0038).
Regulatory approval: On April 23, 2024, the FDA granted accelerated approval to tovorafenib (Ojemda) for patients aged ≥6 months with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion/rearrangement or BRAF V600 mutation. Approval was supported by FIREFLY-1, a multicenter, open-label, single-arm trial (n=76) reporting an overall response rate of 51% and a median duration of response of 13.8 months. Common adverse reactions included rash, hair color changes, fatigue, and viral infection.
Regulatory approval: On April 23, 2024, the FDA approved lutetium Lu 177 dotatate (Lutathera) for pediatric patients aged 12 years and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut NETs. The decision was supported by PK/dosimetry/safety data from the ongoing NETTER-P study and efficacy extrapolation from the adult NETTER-1 trial. A post-marketing requirement will assess long-term safety in adolescents.
Regulatory approval: On April 23, 2024, the FDA granted accelerated approval to tovorafenib (Ojemda) for patients aged ≥6 months with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion/rearrangement or BRAF V600 mutation. Approval was based on overall response rate and duration of response from the single-arm FIREFLY-1 trial; continued approval may depend on confirmatory evidence of clinical benefit.
Regulatory approval: On April 22, 2024, the FDA approved nogapendekin alfa inbakicept-pmln (Anktiva) with Bacillus Calmette-Guérin (BCG) for adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. Approval was supported by the single-arm QUILT-3.032 trial (n=77), which reported a complete response (CR) rate of 62% (95% CI, 51–73) and durable responses in a subset of responders.
Regulatory approval: On April 18, 2024, the FDA approved alectinib (Alecensa) for adjuvant treatment after tumor resection in patients with ALK-positive NSCLC, as detected by an FDA-approved test. Approval was based on the global, randomized, open-label ALINA trial, which showed significantly improved investigator-assessed disease-free survival versus platinum-based chemotherapy in stage II–IIIA and overall stage IB–IIIA populations. Recommended dose is 600 mg orally twice daily with food for 2 years or until recurrence/toxicity.
Regulatory approval: On April 5, 2024, the FDA granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu) for adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumors after prior systemic therapy and with no satisfactory alternative options. The tumor-agnostic indication is based on objective response rate and duration of response from three multicenter trials; continued approval may depend on confirmatory evidence of clinical benefit.
Regulatory approval: On April 5, 2024, the FDA granted accelerated approval to fam-trastuzumab deruxtecan-nxki (Enhertu) for adult patients with unresectable or metastatic HER2-positive solid tumors who have received prior systemic therapy and have no satisfactory alternative treatment options. The decision was supported by objective response rate and duration of response results from three multicenter trials (DESTINY-PanTumor02, DESTINY-Lung01, DESTINY-CRC02). Labeling includes a Boxed Warning for interstitial lung disease and embryo-fetal toxicity.
Regulatory approval: On March 22, 2024, the FDA granted accelerated approval to mirvetuximab soravtansine-gynx (Elahere) for adults with FRα-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after 1–3 prior systemic regimens, with selection by an FDA-approved test. Efficacy supporting the post-marketing requirement came from the randomized MIRASOL (Study 0416) trial, showing improved OS, PFS, and ORR versus investigator’s choice chemotherapy.
Regulatory approval: On March 21, 2024, the FDA approved safety labeling changes for fluorouracil injection products to address the risk of serious adverse reactions in patients with dihydropyrimidine dehydrogenase (DPD) deficiency. Updates were made to the Highlights and full Prescribing Information (Warnings and Precautions; Patient Counseling) and a new Pharmacogenomics subsection was added. The changes align with labeling updates previously approved for capecitabine (Xeloda) in 2022.
Regulatory approval: On March 19, 2024, the FDA granted accelerated approval to ponatinib (Iclusig) with chemotherapy for adult patients with newly diagnosed Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL). Approval was supported by the phase III PhALLCON trial (NCT03589326), where minimal residual disease (MRD)–negative complete remission at end of induction was 30% with ponatinib vs 12% with imatinib plus chemotherapy (risk difference 0.18; p=0.0004).
Regulatory approval: On March 7, 2024, the FDA approved nivolumab plus cisplatin and gemcitabine for first-line treatment of adults with unresectable or metastatic urothelial carcinoma. The decision was based on CHECKMATE-901 (NCT03036098; n=608), which showed statistically significant improvements in overall survival and progression-free survival versus cisplatin/gemcitabine alone. Median OS was 21.7 vs 18.9 months (HR 0.78), and median PFS was 7.9 vs 7.6 months (HR 0.72).
Regulatory approval: On March 7, 2024, the FDA granted accelerated approval to zanubrutinib (Brukinsa) in combination with obinutuzumab for adults with relapsed or refractory follicular lymphoma after ≥2 prior lines of systemic therapy. Approval was supported by the open-label, randomized ROSEWOOD trial (NCT03332017), where overall response rate was 69% with zanubrutinib+obinutuzumab vs 46% with obinutuzumab alone; median duration of response was not reached vs 14.0 months, respectively.
Regulatory approval: On March 6, 2024, the FDA approved inotuzumab ozogamicin (Besponsa; Pfizer) for pediatric patients aged ≥1 year with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia. Approval was based on a multicenter, single-arm, open-label study (n=53) reporting a complete remission (CR) rate of 42% with a median CR duration of 8.2 months; most CRs were MRD-negative by flow cytometry and/or PCR.
Regulatory approval: On March 1, 2024, the FDA approved amivantamab-vmjw (Rybrevant) plus carboplatin and pemetrexed for first-line treatment of locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations (FDA-approved test required). The FDA also granted traditional approval for amivantamab-vmjw monotherapy in adults with the same biomarker whose disease progressed on/after platinum-based chemotherapy, converting a prior accelerated approval. The first-line approval was supported by PAPILLON, showing improved PFS vs chemotherapy alone.
Regulatory approval: On Feb 16, 2024, the FDA granted accelerated approval to lifileucel (Amtagvi), an autologous tumor-derived T-cell immunotherapy, for adults with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody and, if BRAF V600–positive, a BRAF inhibitor with or without a MEK inhibitor. Approval was supported by a global, open-label, single-arm trial; ORR was 31.5% (95% CI 21.1–43.4) with median DoR not reached.
Regulatory approval: On Feb 16, 2024, the FDA approved osimertinib (Tagrisso) in combination with platinum-based chemotherapy for patients with locally advanced or metastatic NSCLC whose tumors harbor EGFR exon 19 deletions or exon 21 L858R mutations (per an FDA-approved test). Approval was supported by the phase 3 FLAURA 2 trial, where osimertinib plus chemotherapy significantly improved investigator-assessed PFS versus osimertinib alone (HR 0.62; median 25.5 vs 16.7 months). OS data were immature.
Regulatory approval: On Feb 15, 2024, the FDA granted traditional approval to tepotinib (Tepmetko) for adults with metastatic non-small cell lung cancer (NSCLC) harboring MET exon 14 skipping alterations. Tepotinib previously received accelerated approval (Feb 3, 2021) based on ORR and DOR from the VISION trial; conversion was supported by additional patients and longer follow-up assessing DOR. ORR and DOR were reviewed by a blinded independent review committee.
Regulatory approval: On Feb 13, 2024, the FDA approved irinotecan liposome (Onivyde) in combination with oxaliplatin, fluorouracil, and leucovorin (NALIRIFOX) for first-line treatment of metastatic pancreatic adenocarcinoma. Approval was supported by the randomized phase 3 NAPOLI 3 trial (N=770), which showed statistically significant improvements in overall survival (median 11.1 vs 9.2 months; HR 0.84) and progression-free survival (median 7.4 vs 5.6 months; HR 0.70) versus gemcitabine plus nab-paclitaxel.
Regulatory approval: The FDA granted accelerated approval (Jan 2024) to erdafitinib (Balversa) for adults with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations (per an FDA-approved companion diagnostic) whose disease progressed on/after ≥1 prior systemic therapy. In the randomized BLC3001 Cohort 1 trial (n=266), erdafitinib improved OS, PFS, and ORR versus docetaxel or vinflunine. The label notes it is not recommended for patients eligible for and untreated with prior PD-1/PD-L1 therapy.
Regulatory approval: On Jan 12, 2024, the FDA approved pembrolizumab (Keytruda) in combination with chemoradiotherapy (CRT) for patients with FIGO 2014 Stage III–IVA cervical cancer. The decision was supported by KEYNOTE-A18 (NCT04221945), which showed a statistically significant progression-free survival (PFS) benefit in the overall population; exploratory subgroup results suggested the benefit was primarily driven by the Stage III–IVA cohort. Overall survival data were not mature at the time of the PFS analysis.
Regulatory approval: On Dec 15, 2023, the FDA approved enfortumab vedotin-ejfv plus pembrolizumab for patients with locally advanced or metastatic urothelial cancer. Efficacy was supported by EV-302/KN-A39, where the combination improved overall survival (median 31.5 vs 16.1 months; HR 0.47) and progression-free survival (median 12.5 vs 6.3 months; HR 0.45) versus platinum-based chemotherapy in previously untreated advanced disease.
Regulatory approval: On Dec 14, 2023, the FDA approved belzutifan (Welireg; Merck) for patients with advanced renal cell carcinoma following prior PD-1/PD-L1 inhibitor and a VEGF tyrosine kinase inhibitor. The decision was supported by LITESPARK-005, which showed a statistically significant progression-free survival benefit versus everolimus (HR 0.75; 95% CI 0.63–0.90; 1-sided p=0.0008). Overall survival was immature at the time of analysis.
Regulatory approval: On Dec 13, 2023, the FDA approved eflornithine (IWILFIN; USWM, LLC) to reduce relapse risk in adult and pediatric patients with high-risk neuroblastoma who achieved at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Approval was supported by an externally controlled comparison of Study 3b versus a propensity score–matched external control from ANBL0032, with EFS HR 0.48 and OS HR 0.32 in the primary analysis; uncertainty remains due to the study design.
Regulatory approval: On Dec 1, 2023, the FDA granted accelerated approval to pirtobrutinib (Jaypirca) for adults with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor. Approval was supported by the single-arm BRUIN trial (n=108), with an independent review committee–assessed ORR of 72% and median duration of response of 12.2 months; all responses were partial.
Regulatory approval: On Nov 27, 2023, the FDA approved nirogacestat (OGSIVEO) for adult patients with progressing desmoid tumors who require systemic treatment, described as the first approved treatment for desmoid tumors. Approval was supported by the randomized, double-blind, placebo-controlled DeFi trial (n=142) in patients with progressing desmoid tumors not amenable to surgery. Median PFS was not reached with nirogacestat vs 15.1 months with placebo; ORR was 41% vs 8%.
Regulatory approval: On Nov 27, 2023, the FDA approved nirogacestat (OGSIVEO; SpringWorks Therapeutics) for adult patients with progressing desmoid tumors who require systemic treatment. The decision was supported by the phase 3 DeFi trial (NCT03785964), which reported improved progression-free survival versus placebo (HR 0.29; p<0.001) and higher objective response rate (41% vs 8%; p<0.001). This is described as the first approved treatment for desmoid tumors.
Regulatory approval: On Nov 16, 2023 (FDA posting date Nov 17), the FDA approved enzalutamide (Xtandi) for non-metastatic castration-sensitive prostate cancer with biochemical recurrence at high risk for metastasis. Approval was supported by the phase 3 EMBARK trial (NCT02319837), which showed statistically significant improvements in metastasis-free survival for enzalutamide plus leuprolide and for enzalutamide monotherapy versus placebo plus leuprolide. Overall survival data were immature at the time of analysis.
Regulatory approval: On Nov 16, 2023, the FDA approved pembrolizumab plus fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma. Approval was supported by KEYNOTE-859 (n=1579), which showed statistically significant improvements in overall survival, progression-free survival, and overall response rate versus placebo plus chemotherapy. Recommended dosing is 200 mg every 3 weeks or 400 mg every 6 weeks.
Regulatory approval: On Nov 16, 2023, the FDA approved capivasertib (Truqap) plus fulvestrant for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer with ≥1 PIK3CA/AKT1/PTEN alteration (FDA-approved test), after progression on at least one endocrine-based regimen in the metastatic setting or recurrence on/within 12 months of completing adjuvant therapy. FDA also approved FoundationOne®CDx as a companion diagnostic. Approval was supported by CAPItello-291 PFS results.
Regulatory approval: On Nov 15, 2023, the FDA approved repotrectinib (Augtyro) for locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). The approval includes both ROS1 TKI–naïve patients and patients previously treated with a ROS1 tyrosine kinase inhibitor. Approval was based on the single-arm TRIDENT-1 trial, with confirmed ORR 79% in TKI-naïve patients and 38% in prior ROS1 inhibitor–treated patients; median DOR was 34.1 and 14.8 months, respectively.
Regulatory approval: On Nov 15, 2023, the FDA approved repotrectinib (Augtyro) for locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC). The approval includes patients who are ROS1 tyrosine kinase inhibitor (TKI)–naïve and those previously treated with a ROS1 TKI. The decision was based on the single-arm TRIDENT-1 trial, with confirmed ORR 79% in TKI-naïve patients and 38% after a prior ROS1 inhibitor; median duration of response was 34.1 and 14.8 months, respectively.
Regulatory approval: On Nov 8, 2023, the FDA approved fruquintinib (Fruzaqla) for adults with metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type and medically appropriate, an anti-EGFR therapy. Approval was supported by FRESCO-2 and FRESCO, where overall survival favored fruquintinib vs placebo. Reviews at other agencies are ongoing under Project Orbis.
Regulatory approval: On Nov 8, 2023, the FDA approved fruquintinib (Fruzaqla; Takeda) for adults with metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF therapy, and (if RAS wild-type and medically appropriate) an anti-EGFR therapy. Approval was supported by OS results from the randomized, placebo-controlled FRESCO-2 and FRESCO trials. Common adverse reactions included hypertension and palmar-plantar erythrodysesthesia.
Regulatory approval news: On Nov 7, 2023, the FDA revised the accelerated approval indication for pembrolizumab (Keytruda) given with trastuzumab plus fluoropyrimidine- and platinum-containing chemotherapy as first-line treatment for locally advanced unresectable or metastatic HER2-positive gastric/GEJ adenocarcinoma. Use is now restricted to tumors with PD-L1 expression (CPS ≥1) determined by an FDA-approved test. FDA also approved Agilent PD-L1 IHC 22C3 pharmDx as a companion diagnostic.
Regulatory approval: On Oct 31, 2023, the FDA approved pembrolizumab (Keytruda) with gemcitabine and cisplatin for locally advanced unresectable or metastatic biliary tract cancer. The decision was supported by KEYNOTE-966 (NCT04003636), which enrolled 1,069 patients without prior systemic therapy for advanced disease. Pembrolizumab plus chemotherapy improved overall survival versus placebo plus chemotherapy (HR 0.83; median OS 12.7 vs 10.9 months).
Regulatory approval: On Oct 27, 2023, the FDA approved toripalimab-tpzi (LOQTORZI) for adults with nasopharyngeal carcinoma. It is approved with cisplatin and gemcitabine for first-line treatment of metastatic or recurrent, locally advanced disease, and as monotherapy for recurrent unresectable or metastatic disease progressing on or after platinum chemotherapy. Approval was supported by JUPITER-02 (PFS and OS benefit) and POLARIS-02 (ORR and DOR).
Regulatory approval: On Oct 24, 2023, the FDA approved ivosidenib (Tibsovo) for adult patients with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible IDH1 mutation detected by an FDA-approved test. The Abbott RealTime IDH1 Assay was also approved as a companion diagnostic. Approval was based on the single-arm AG120-C-001 trial (n=18), with a complete remission rate of 38.9%. The label includes a Boxed Warning for differentiation syndrome.
Regulatory approval: On Oct 24, 2023, the FDA approved ivosidenib (Tibsovo) for adults with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible IDH1 mutation detected by an FDA-approved test. Approval was based on the open-label, single-arm AG120-C-001 study (n=18). The FDA also approved the Abbott RealTime IDH1 Assay as a companion diagnostic. Boxed Warning highlights potentially fatal differentiation syndrome; QTc prolongation is also noted.
Regulatory approval: On Oct 20, 2023, the FDA granted accelerated approval to entrectinib (Rozlytrek) for pediatric patients >1 month old with NTRK gene fusion–positive solid tumors (no known acquired resistance mutation) that are metastatic or where resection would likely cause severe morbidity, and that have progressed after treatment or lack satisfactory standard therapy. The FDA also approved a new oral pellet formulation and added instructions for preparing an oral suspension from capsules.
Regulatory approval: On Oct 16, 2023, the FDA approved pembrolizumab (Keytruda) with platinum-containing chemotherapy as neoadjuvant therapy, followed by continuation of single-agent pembrolizumab as adjuvant therapy after surgery, for resectable NSCLC (tumors ≥4 cm or node positive). The decision was based on KEYNOTE-671, which showed improvements in OS and investigator-assessed EFS versus placebo plus chemotherapy using the same perioperative approach.
Regulatory approval: On Oct 13, 2023, the FDA approved nivolumab (Opdivo) for adjuvant treatment of completely resected Stage IIB/C melanoma in patients aged ≥12 years. Approval was supported by the randomized, double-blind CHECKMATE-76K trial (N=790), in which nivolumab reduced the risk of recurrence or death versus placebo (RFS HR 0.42; 95% CI 0.30–0.59; p<0.0001); median RFS was not reached in either arm. Common adverse reactions (≥20%) included fatigue, musculoskeletal pain, rash, diarrhea, and pruritis.
Regulatory approval: On Oct 11, 2023, the FDA approved encorafenib plus binimetinib for adult patients with metastatic non-small cell lung cancer (NSCLC) harboring a BRAF V600E mutation detected by an FDA-approved test. Approval was supported by the single-arm PHAROS study (n=98). In treatment-naïve patients, ORR was 75% and median DoR was not estimable; in previously treated patients, ORR was 46% with median DoR 16.7 months.
Regulatory approval: On September 26, 2023, the FDA approved bosutinib (Bosulif, Pfizer) for pediatric patients aged ≥1 year with chronic-phase (CP) Philadelphia chromosome–positive (Ph+) chronic myelogenous leukemia (CML) that is newly diagnosed or resistant/intolerant to prior therapy. The agency also approved a new capsule dosage form (50 mg and 100 mg). Approval was supported by results from the multicenter, nonrandomized, open-label BCHILD trial (NCT04258943).
Regulatory approval: On Sept 14, 2023, the FDA approved updated labeling for temozolomide (Temodar, Merck) under Project Renewal, an Oncology Center of Excellence initiative to modernize labeling for older oncology drugs. The update includes new/revised indications in adult anaplastic astrocytoma and retains the adult newly diagnosed glioblastoma indication. Labeling changes also revise dosing regimens and add capsule-handling risk information.
Regulatory approval: On August 14, 2023, the FDA approved HEPZATO KIT (melphalan for Injection/Hepatic Delivery System; Delcath Systems) as a liver-directed treatment for adult patients with uveal melanoma and unresectable hepatic metastases involving <50% of the liver, with no extrahepatic disease or limited extrahepatic disease amenable to resection or radiation. Approval was supported by the single-arm FOCUS trial (NCT02678572) reporting an independently reviewed ORR of 36.3% and median DoR of 14 months. The product has Boxed Warnings and a REMS.
Regulatory approval: On Aug 14, 2023, the FDA granted accelerated approval to elranatamab-bcmm (Elrexfio), a BCMA-directed CD3 T-cell engager, for adults with relapsed/refractory multiple myeloma after ≥4 prior lines including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. Approval was supported by the single-arm MagnetisMM-3 trial. In 97 BCMA-naïve patients at the recommended dose, ORR was 57.7% and median DOR was not reached at 11.1 months’ median follow-up among responders.
Regulatory approval: On Aug 11, 2023, the FDA approved the fixed-dose combination niraparib/abiraterone acetate (Akeega) with prednisone for adults with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC), as determined by an FDA-approved test. Approval was supported by Cohort 1 of the phase 3 MAGNITUDE trial, where BRCA-mutated patients had improved radiographic PFS versus placebo plus abiraterone/prednisone; OS findings were exploratory.
Regulatory approval: On Aug 9, 2023, the FDA granted accelerated approval to talquetamab-tgvs (Talvey) for adults with relapsed or refractory multiple myeloma after at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Approval was supported by the single-arm MonumenTAL-1 study, with ORR ~73% across two dosing schedules and median DOR 9.5 months (weekly dose) or not estimable (biweekly dose).
Regulatory approval: The FDA granted accelerated approval to talquetamab-tgvs (Talvey) on Aug 9, 2023, and to elranatamab-bcmm (Elrexfio) on Aug 14, 2023, for adults with relapsed or refractory multiple myeloma after ≥4 prior lines including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Both labels carry Boxed Warnings for cytokine release syndrome (CRS) and neurologic toxicity/ICANS and are subject to REMS programs.
Regulatory approval: On Aug 9, 2023, the FDA granted regular approval to pralsetinib (Gavreto) for adult patients with metastatic RET fusion–positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test. Pralsetinib previously received accelerated approval for this indication in 2020. The regular approval was supported by additional ARROW trial data and longer follow-up assessing durability of response, with ORR and DOR as key endpoints.
Regulatory approval: On Aug 2, 2023, the FDA approved trifluridine/tipiracil (LONSURF) in combination with bevacizumab for previously treated metastatic colorectal cancer. The decision was supported by the randomized SUNLIGHT trial (NCT04737187), in which LONSURF plus bevacizumab improved overall survival versus LONSURF alone (HR 0.61; median 10.8 vs 7.5 months) and progression-free survival (HR 0.44; median 5.6 vs 2.4 months).
Regulatory approval: On July 31, 2023, the FDA approved dostarlimab-gxly (Jemperli) with carboplatin and paclitaxel, followed by single-agent dostarlimab-gxly, for primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR) as determined by an FDA-approved test, or microsatellite instability–high (MSI-H). The approval was supported by the RUBY trial subgroup (n=122 dMMR/MSI-H), where investigator-assessed PFS improved versus placebo-containing regimens (median 30.3 vs 7.7 months; HR 0.29).
Regulatory approval: On July 20, 2023, the FDA approved quizartinib (Vanflyta) in combination with standard cytarabine/anthracycline induction and cytarabine consolidation, and as post-consolidation maintenance monotherapy, for adults with newly diagnosed FLT3-ITD–positive AML as detected by an FDA-approved test. The companion diagnostic LeukoStrat CDx FLT3 Mutation Assay was also approved. Approval was supported by QuANTUM-First, which showed an overall survival benefit versus placebo (HR 0.78; 95% CI 0.62–0.98).
Regulatory approval: On June 20, 2023, the FDA approved talazoparib (Talzenna) in combination with enzalutamide for patients with homologous recombination repair (HRR) gene–mutated metastatic castration-resistant prostate cancer (mCRPC). Approval was supported by the randomized, double-blind, placebo-controlled TALAPRO-2 trial, which showed a statistically significant improvement in radiographic progression-free survival (rPFS) for talazoparib plus enzalutamide versus placebo plus enzalutamide.
Regulatory approval: On June 15, 2023 (announced June 16), the FDA granted accelerated approval to glofitamab-gxbm (Columvi) for relapsed or refractory diffuse large B-cell lymphoma (DLBCL, NOS) or large B-cell lymphoma arising from follicular lymphoma after ≥2 prior systemic therapies. Approval was supported by the single-arm NP30179 study, which reported a 56% objective response rate with 43% complete responses and an estimated median duration of response of 18.4 months among responders. Labeling includes a Boxed Warning for cytokine release syndrome.
Regulatory approval: On May 31, 2023, the FDA approved olaparib (Lynparza) in combination with abiraterone and prednisone (or prednisolone) for adult patients with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC), as determined by an FDA-approved companion diagnostic. Approval was supported by the PROpel trial, including an exploratory BRCA-mutated subgroup analysis showing improved rPFS versus placebo plus abiraterone.
Regulatory approval: On May 31, 2023, the FDA approved olaparib (Lynparza) in combination with abiraterone and prednisone (or prednisolone) for adult patients with deleterious or suspected deleterious BRCA-mutated metastatic castration-resistant prostate cancer (mCRPC), as determined by an FDA-approved companion diagnostic. The approval was based on PROpel, which showed a statistically significant radiographic progression-free survival benefit in the intent-to-treat population, with exploratory analyses suggesting the benefit was primarily driven by BRCA-mutated disease.
Regulatory approval news: On May 19, 2023, the FDA granted accelerated approval to epcoritamab-bysp (Epkinly), a bispecific CD20-directed CD3 T-cell engager, for adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma) and high-grade B-cell lymphoma after ≥2 prior lines of systemic therapy. Approval was based on overall response rate and duration of response from the single-arm EPCORE NHL-1 trial. The label carries a Boxed Warning for CRS and ICANS.
Regulatory approval: On May 19, 2023, the FDA granted accelerated approval to epcoritamab-bysp (Epkinly), a CD20xCD3 bispecific T-cell engager, for relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma) and high-grade B-cell lymphoma after ≥2 prior lines of systemic therapy. Approval was supported by the single-arm EPCORE NHL-1 trial, which reported an overall response rate of 61% and complete responses in 38% of patients.
Regulatory approval: On April 19, 2023, the FDA approved polatuzumab vedotin-piiq (Polivy) with rituximab, cyclophosphamide, doxorubicin, and prednisone for adults with previously untreated diffuse large B-cell lymphoma (not otherwise specified) or high-grade B-cell lymphoma with an International Prognostic Index score ≥2. Approval was based on the POLARIX trial (n=879), in which substituting polatuzumab for vincristine improved investigator-assessed progression-free survival (HR 0.73); overall survival was not significantly different.
Regulatory approval: On April 19, 2023, the FDA approved polatuzumab vedotin-piiq (Polivy) in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone (R-CHP) for adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL), not otherwise specified, or high-grade B-cell lymphoma (HGBL) with International Prognostic Index (IPI) score ≥2. Approval was based on the phase 3 POLARIX trial showing improved investigator-assessed progression-free survival versus R-CHOP.
Regulatory approval: On April 17, 2023, the FDA approved omidubicel-onlv (Omisirge; Gamida Cell Ltd.) for adults and pediatric patients ≥12 years with hematologic malignancies planned for umbilical cord blood transplantation after myeloablative conditioning. The approval is to reduce time to neutrophil recovery and the incidence of infection. The decision was supported by the open-label, multicenter, randomized Study P0501 (NCT02730299) comparing omidubicel-onlv transplantation with unmanipulated cord blood transplantation.
Regulatory approval: On April 17, 2023, the FDA approved omidubicel-onlv (Omisirge) for adults and pediatric patients aged ≥12 years with hematologic malignancies planned for umbilical cord blood transplantation after myeloablative conditioning, to reduce time to neutrophil recovery and incidence of infection. Approval was supported by Study P0501, which reported median neutrophil recovery of 12 vs 22 days (omidubicel-onlv vs unmanipulated cord blood) and Day-100 specified infection rates of 39% vs 60%.
Regulatory approval: On April 3, 2023, the FDA granted accelerated approval to enfortumab vedotin-ejfv (Padcev) in combination with pembrolizumab (Keytruda) for patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy. The decision was supported by EV-103/KEYNOTE-869 (multi-cohort; some single-arm, Cohort K randomized). In 121 treated patients, confirmed ORR was 68% (12% CR); median DOR was 22 months in dose-escalation+Cohort A and not reached in Cohort K.
Regulatory approval: On April 3, 2023, the FDA granted accelerated approval to enfortumab vedotin-ejfv plus pembrolizumab for patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin-containing chemotherapy. The decision was supported by EV-103/KEYNOTE-869 (NCT03288545), where 121 patients received the combination. Confirmed ORR was 68% (12% complete responses) by blinded independent central review using RECIST v1.1; median duration of response was 22 months in dose escalation+Cohort A and not reached in Cohort K.
Regulatory approval: On March 22, 2023, the US FDA granted accelerated approval to retifanlimab-dlwr (Zynyz) for adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma. The decision was based on PODIUM-201, an open-label, multiregional, single-arm study in 65 previously untreated patients with advanced disease. Independent central review reported an objective response rate of 52% (18% complete responses), with durable responses observed in subsets at ≥6 and ≥12 months.
Regulatory approval: On March 22, 2023, the FDA granted accelerated approval to retifanlimab-dlwr (Zynyz; Incyte) for adults with metastatic or recurrent locally advanced Merkel cell carcinoma. The decision was based on PODIUM-201 (NCT03599713), a single-arm study in 65 previously untreated patients with advanced disease, reporting an objective response rate of 52% (95% CI, 40–65) and complete responses in 18%.
Regulatory approval: On March 16, 2023, the FDA approved dabrafenib (Tafinlar) with trametinib (Mekinist) for pediatric patients aged ≥1 year with low-grade glioma harboring a BRAF V600E mutation who require systemic therapy. The FDA also approved new oral formulations of both agents for patients unable to swallow pills. Efficacy was evaluated in Study CDRB436G2201, an open-label randomized trial comparing dabrafenib+trametinib versus carboplatin+vincristine, with ORR by independent review as the major endpoint.
Regulatory approval: On March 16, 2023, the FDA approved dabrafenib plus trametinib for pediatric patients aged ≥1 year with low-grade glioma (WHO grade 1–2) harboring a BRAF V600E mutation who require systemic therapy. The agency also approved new oral formulations for patients unable to swallow pills. Approval was supported by Study CDRB436G2201, where dabrafenib/trametinib improved ORR and PFS versus carboplatin/vincristine; interim OS was not statistically significant.
Regulatory approval: On March 3, 2023, the FDA approved abemaciclib (Verzenio) with endocrine therapy (tamoxifen or an aromatase inhibitor) for adjuvant treatment of adult patients with HR-positive, HER2-negative, node-positive early breast cancer at high risk of recurrence. The approval removes the prior Ki-67 ≥20% testing requirement and restricts the indication to patients meeting cohort 1 high-risk clinicopathologic criteria from monarchE.
Regulatory approval: On March 3, 2023, the FDA approved abemaciclib (Verzenio) with endocrine therapy (tamoxifen or an aromatase inhibitor) for adjuvant treatment of adults with HR-positive, HER2-negative, node-positive early breast cancer at high risk of recurrence. The expanded indication removes the prior requirement for Ki-67 ≥20% testing and is restricted to patients meeting cohort 1 high-risk criteria from monarchE. Benefit was shown in invasive disease-free survival; overall survival remains immature.
Regulatory approval news: On Feb 9, 2023, the FDA approved dostarlimab-gxly (Jemperli) for adults with mismatch repair–deficient (dMMR) recurrent or advanced endometrial cancer, determined by an FDA-approved test, after progression on/after a prior platinum-containing regimen and not candidates for curative surgery or radiation. Regular approval was supported by the single-arm GARNET trial (NCT02715284), with confirmed ORR 45.4% and median duration of response not reached.
Regulatory approval: On Feb 9, 2023, the FDA approved dostarlimab-gxly (Jemperli) for adults with mismatch repair–deficient (dMMR) recurrent or advanced endometrial cancer, determined by an FDA-approved test, that progressed on/after a prior platinum-containing regimen in any setting and who are not candidates for curative surgery or radiation. Efficacy was evaluated in the GARNET trial; confirmed ORR was 45.4% with median duration of response not reached.
Regulatory approval: On Feb 3, 2023, the FDA approved sacituzumab govitecan-hziy (Trodelvy) for patients with unresectable locally advanced or metastatic hormone receptor–positive, HER2-negative breast cancer after endocrine-based therapy and at least two additional systemic therapies in the metastatic setting. Approval was supported by TROPiCS-02 (n=543), where median PFS was 5.5 vs 4.0 months and median OS was 14.4 vs 11.2 months versus single-agent chemotherapy.
Regulatory approval: On Feb 3, 2023, the FDA approved sacituzumab govitecan-hziy (Trodelvy) for unresectable locally advanced or metastatic HR-positive, HER2-negative breast cancer after endocrine-based therapy and at least two additional systemic therapies in the metastatic setting. Approval was supported by the phase 3 TROPiCS-02 trial, which showed improved PFS and OS versus investigator’s choice single-agent chemotherapy.
Regulatory news: On Jan 27, 2023, the FDA granted accelerated approval to pirtobrutinib (Jaypirca) for relapsed or refractory mantle cell lymphoma after ≥2 prior systemic therapies including a BTK inhibitor, based on the single-arm BRUIN trial (ORR 50%, CR 13%, median DOR 8.3 months). The FDA also approved elacestrant (Orserdu) for postmenopausal women or adult men with ER-positive, HER2-negative, ESR1-mutated advanced/metastatic breast cancer after ≥1 endocrine therapy line, supported by EMERALD (ESR1-mutated median PFS 3.8 vs 1.9 months).
Regulatory approval: On Jan 27, 2023, the FDA granted accelerated approval to pirtobrutinib (Jaypirca; Eli Lilly) for adults with relapsed or refractory mantle cell lymphoma after at least two prior lines of systemic therapy, including a BTK inhibitor. Approval was based on the single-arm BRUIN trial in BTK inhibitor–pretreated MCL (n=120), showing an overall response rate of 50% (13% complete responses) and an estimated median duration of response of 8.3 months.
Regulatory approval: On Jan 27, 2023, the FDA approved elacestrant (Orserdu) for postmenopausal women or adult men with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer after progression on at least one line of endocrine therapy. Approval was supported by the randomized EMERALD trial, which showed a progression-free survival benefit in the ESR1-mutated subgroup. The FDA also approved Guardant360 CDx as a companion diagnostic to identify eligible patients.
Regulatory approval: On January 26, 2023, the FDA approved pembrolizumab (Keytruda) as adjuvant treatment for adult patients with stage IB, II, or IIIA non-small cell lung cancer following complete resection and platinum-based chemotherapy. The indication was limited to patients who received prior adjuvant chemotherapy. Approval was based on KEYNOTE-091, which met its primary endpoint of investigator-assessed disease-free survival in the overall population; an exploratory subgroup without adjuvant chemotherapy showed a disease-free survival hazard ratio of 1.25.
Regulatory approval: On Jan 26, 2023, the FDA approved pembrolizumab (Keytruda) as adjuvant treatment after complete resection and platinum-based chemotherapy for stage IB (T2a ≥4 cm), II, or IIIA NSCLC. Approval was supported by KEYNOTE-091, which met its primary endpoint of improved investigator-assessed disease-free survival (DFS) in the overall population. In chemotherapy-treated patients, median DFS was 58.7 vs 34.9 months (HR 0.73).
Regulatory approval news: On Jan 19, 2023, the FDA granted accelerated approval to tucatinib (Tukysa) plus trastuzumab for RAS wild-type, HER2-positive unresectable or metastatic colorectal cancer after progression on fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. Approval was supported by the open-label MOUNTAINEER trial (n=84), with ORR 38% (95% CI 28–49) and median DOR 12.4 months (95% CI 8.5–20.5) by blinded independent central review.
Regulatory approval: On January 19, 2023, the FDA approved zanubrutinib (Brukinsa; BeiGene USA, Inc.) for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The approval was supported by efficacy results from the SEQUOIA trial in treatment-naïve CLL/SLL and the ALPINE trial in relapsed/refractory CLL/SLL, with key outcomes including progression-free survival and response rates as assessed by independent review.
Regulatory approval news: On Jan 19, 2023, the FDA granted accelerated approval to tucatinib (Tukysa) plus trastuzumab for RAS wild-type, HER2-positive unresectable or metastatic colorectal cancer after progression on fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. The FDA also approved zanubrutinib (Brukinsa) for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), supported by SEQUOIA and ALPINE trial results.
Regulatory approval: On Dec 22, 2022, the FDA granted accelerated approval to mosunetuzumab-axgb (Lunsumio), a bispecific CD20-directed CD3 T-cell engager, for adults with relapsed or refractory follicular lymphoma after ≥2 lines of systemic therapy. Approval was based on the open-label GO29781 study (n=90 efficacy population), with an independently assessed objective response rate of 80% (60% complete response). The label includes a Boxed Warning for serious or life-threatening cytokine release syndrome.
Regulatory approval: On December 16, 2022, the FDA approved nadofaragene firadenovec-vncg (Adstiladrin) for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non–muscle invasive bladder cancer with carcinoma in situ (CIS), with or without papillary tumors. Approval was supported by Study CS-003, a multicenter single-arm trial. In the CIS evaluable cohort, complete response (CR) at any time was 51%, median duration of response was 9.7 months, and 46% of responders maintained CR for at least 1 year.
Regulatory approval: On Dec 14, 2022, the FDA approved updated labeling for capecitabine tablets (Xeloda) under the Oncology Center of Excellence’s Project Renewal pilot program. The revised label includes new and updated indications across colorectal, rectal, breast, gastric/GEJ/esophageal, and pancreatic cancers, along with updated dosing and safety information. The update also revises sections on renal impairment, exposure risks from crushed tablets, dihydropyrimidine dehydrogenase deficiency, clinical pharmacology, and patient counseling materials.
Regulatory approval news: In an FDA D.I.S.C.O. Burst Edition update, the FDA reported approvals in December 2022 for two oncology agents. On Dec 9, 2022, atezolizumab (Tecentriq) was approved for unresectable or metastatic alveolar soft part sarcoma in adults and pediatric patients aged ≥2 years, supported by a single-arm study (ORR 24%). On Dec 12, 2022, the FDA granted accelerated approval to adagrasib (Krazati) for previously treated KRAS G12C–mutated locally advanced/metastatic NSCLC (ORR 43%; median DOR 8.5 months).
Regulatory approval: On Dec 1, 2022, the FDA approved olutasidenib (Rezlidhia) capsules for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation detected by an FDA-approved test. The approval was supported by Study 2102-HEM-101, an open-label, single-arm, multicenter trial (n=147). Key endpoints included CR+CRh rate (35%) and transfusion independence metrics. Labeling includes a Boxed Warning for potentially fatal differentiation syndrome.
Regulatory approval: On Nov 14, 2022, the FDA granted accelerated approval to mirvetuximab soravtansine-gynx (Elahere) for adults with folate receptor alpha (FRα)–positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after 1–3 prior systemic regimens. Approval was supported by the single-arm Study 0417 (n=106), with confirmed ORR 31.7% and median DOR 6.9 months in the efficacy-evaluable population. The FDA also approved the VENTANA FOLR1 RxDx Assay as a companion diagnostic.