FDA approves erdafitinib for locally advanced or metastatic urothelial carcinoma

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.

FDA approval (accelerated): erdafitinib in FGFR3-altered advanced urothelial carcinoma

On January 19, 2024, the FDA granted accelerated approval to erdafitinib (Balversa; Janssen Biotech) for adult patients with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic, whose disease has progressed on or after at least one line of prior systemic therapy. The label states erdafitinib is not recommended for patients eligible for and who have not received prior PD-1/PD-L1 inhibitor therapy. This action amends a prior accelerated approval that covered susceptible FGFR3 or FGFR2 alterations after prior platinum-containing chemotherapy.

Key efficacy evidence

Efficacy was evaluated in Study BLC3001 Cohort 1, a randomized, open-label trial (n=266) in mUC with selected FGFR3 alterations after 1–2 prior systemic treatments, including a PD-1/PD-L1 inhibitor, comparing erdafitinib vs investigator’s choice chemotherapy (docetaxel or vinflunine).

  • Overall survival (primary): median 12.1 vs 7.8 months (HR 0.64, 95% CI 0.47–0.88; p=0.0050)
  • PFS: median 5.6 vs 2.7 months (HR 0.58; p=0.0002)
  • Confirmed ORR: 35.3% vs 8.5% (p<0.001)

Safety and dosing (label highlights)

Common (>20%) adverse reactions/lab abnormalities included increased phosphate, nail disorders, diarrhea, stomatitis, fatigue, and others. Recommended starting dose: 8 mg orally once daily, with potential increase to 9 mg based on tolerability at days 14–21.

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