The FDA announced accelerated approval of erdafitinib (Balversa; Janssen Pharmaceutical), the first fibroblast growth factor receptor (FGFR) kinase inhibitor for adults with locally advanced bladder cancer with a FGFR3 mutation or FGFR2 fusion who have progressed despite platinum-based chemotherapy.

In a trial of 87 patients, 2.3% had a complete response and 29.9% had a partial response to the once-daily oral therapy that lasted a median 5.4 months. A quarter of patients did not respond to prior PD-L1/PD-1 treatment.

“Today’s approval represents the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a news release. “FGFRs regulate important biological processes including cell growth and division during development and tissue repair. This drug works by targeting genetic alterations in FGFRs.”

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The most common side effects of the drug were increased phosphate, mouth sores, fatigue, increased creatinine, diarrhea, dry mouth, nail malformation, increased alanine aminotransferase and alkaline phosphatase, low sodium, decreased appetite, altered taste, anemia, dry skin, dry eyes, and hair loss. Other side effects include redness, swelling, hand foot syndrome, constipation, stomach pain, nausea, and muscle pain.

Erdafitinib may cause inflammation in the eye, so patients are advised to have periodic eye examinations. Women who are pregnant or breastfeeding should not take the drug, the FDA warned.

The FDA simultaneously approved a companion diagnostic for use with erdafitinib, the QIAGEN therascreen® FGFR RGQ Reverse-transcription (RT)-polymerase chain reaction (PCR) Kit, according to Janssen.


FDA approves first targeted therapy for metastatic bladder cancer (news release). FDA, April 12, 2019.

BALVERSA™ (erdafitinib) Receives U.S. FDA approval for the treatment of patients with locally advanced or metastatic urothelial carcinoma with certain FGFR genetic alterations (press release). Horsham, PA. Janssen Pharmaceutical Companies of Johnson & Johnson; April 12, 2019.