U.S. flag An official website of the United States government
  1. Home
  2. Drugs
  3. Development & Approval Process | Drugs
  4. Drug Approvals and Databases
  5. Resources for Information on Approved Drugs
  6. FDA grants accelerated approval to erdafitinib for metastatic urothelial carcinoma
  1. Resources for Information on Approved Drugs

FDA grants accelerated approval to erdafitinib for metastatic urothelial carcinoma

On April 12, 2019, the Food and Drug Administration granted accelerated approval to erdafitinib (BALVERSA, Janssen Pharmaceutical Companies) for patients with locally advanced or metastatic urothelial carcinoma, with susceptible FGFR3 or FGFR2 genetic alterations, that has progressed during or following platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

Patients should be selected for therapy based on an FDA-approved companion diagnostic for erdafitinib. Today, the FDA also approved the therascreen® FGFR RGQ RT-PCR Kit, developed by QIAGEN, for use as a companion diagnostic for this therapeutic indication.

Erdafitinib approval was based on data from a cohort of 87 patients enrolled on Study BLC2001 (NCT02365597), a multicenter, open-label, single-arm trial. These patients had locally advanced or metastatic urothelial carcinoma that had progressed on or after at least one prior chemotherapy and had certain FGFR3 gene mutations or FGFR2 or FGFR3 gene fusions. Patients received erdafitinib at a starting dose of 8 mg once daily with a dose increase to 9 mg daily in those whose serum phosphate levels were below the target of 5.5 mg/dL, between days 14 and 17. The starting dose was increased to 9 mg daily in 41% of the patients. Erdafitinib was administered until disease progression or unacceptable toxicity.

The major efficacy outcome measure was objective response rate (ORR) as determined by blinded independent review committee according to RECIST 1.1. ORR was 32.2% (95% CI:22.4, 42.0), with complete responses in 2.3% and partial responses in 29.9%. Median response duration was 5.4 months (95% CI: 4.2, 6.9). Responders included patients who had previously not responded to anti PD-L1 or PD-1 treatment.

Erdafitinib can cause ocular disorders. Central serous retinopathy or retinal pigment epithelial detachment resulting in visual field defect was reported in 25% of patients. The most common adverse reactions reported in at least 40% of patients were increased serum phosphate, stomatitis, fatigue, increased serum creatinine, diarrhea, dry mouth, onycholysis, increased alanine aminotransferase, increased alkaline phosphatase, and decreased sodium.

The recommended initial dose of erdafitinib is 8 mg orally once daily (with or without food) with a dose increase to 9 mg daily if criteria are met (see above).

View full prescribing information for BALVERSA

This indication is approved under accelerated approval based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. FDA granted this application priority review and breakthrough therapy designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

Follow the Oncology Center of Excellence on Twitter @FDAOncology.

Check out recent approvals at the OCE’s podcast, Drug Information Soundcast in Clinical Oncology (D.I.S.C.O.).