FDA grants traditional approval to selpercatinib for locally advanced or metastatic RET fusion-positive solid tumors
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.
FDA decision (regulatory approval)
On July 14, 2026, the FDA granted traditional approval to selpercatinib (Retevmo; Eli Lilly and Company) for adult and pediatric patients aged ≥2 years with locally advanced or metastatic solid tumors harboring a RET gene fusion (as detected by an FDA-approved test) that have progressed on or after prior systemic treatment or for whom no satisfactory alternative treatment options exist. Selpercatinib previously received accelerated approval for this indication in adults (2022) and pediatric patients ≥2 years (2024).
Supporting evidence
Efficacy was evaluated in LIBRETTO-001 (NCT03157128; multicenter, open-label, multi-cohort) and supported by results in RET fusion–positive NSCLC and RET fusion–positive thyroid cancer. In 75 patients with RET fusion–positive tumors other than NSCLC and thyroid cancer, the ORR was 47% (95% CI, 35–59) and median DOR was 24.5 months (95% CI, 11.2–49.1). Reported responding tumor types included colorectal, pancreatic adenocarcinoma, salivary, soft tissue sarcoma, cholangiocarcinoma, skin carcinoma, unknown primary, breast, bronchial carcinoid, ovarian, small intestine, and neuroendocrine tumors (including pancreatic).
In LIBRETTO-121 (NCT03899792), responses were observed in select pediatric/young adult patients with refractory RET fusion–positive solid tumors.
Safety and dosing (label highlights)
Warnings/precautions include hepatotoxicity, ILD/pneumonitis, hypertension, QT prolongation, hemorrhagic events, hypersensitivity, tumor lysis syndrome, impaired wound healing, hypothyroidism, embryo-fetal toxicity, and pediatric slipped capital/upper femoral epiphysis. Adult/adolescent (≥12 years) dosing by weight: <50 kg: 120 mg PO BID; ≥50 kg: 160 mg PO BID.