FDA Accelerates Approval of Targeted Drug for Locally Advanced or Metastatic Bladder Cancer

The FDA has granted accelerated approval to erdafitinib (Balversa; Janssen) for the treatment of adults with locally advanced or metastatic bladder cancer that is positive for the FGFR3 or FGFR2 mutation, and that progressed during or after platinum-containing chemotherapy. Use of erdafitinib, which was granted Breakthrough Therapy designation by the FDA, should be decided based on results from an FDA-approved companion diagnostic device.

“We’re in an era of more personalized or precision medicine, and the ability to target cancer treatment to a patient’s specific genetic mutation or biomarker is becoming the standard, with advances being made in new disease types. Today’s approval represents the first personalized treatment targeting susceptible FGFR genetic alternations for patients with metastatic bladder cancer,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence, in a press statement.

“FGFRs regulate important biological processes including cell growth and division during development and tissue repair. This drug works by targeting genetic alterations in FGFRs,” he added.

The efficacy of erdafitinib was determined in a clinical trial of 87 patients with locally advanced or metastatic bladder cancer and FGFR3 or FGFR2 genetic alterations that progressed after chemotherapy. The overall response rate in these patients was 32.2% (complete response, 2.3%; partial response, approximately 30%) erdafitinib.

On average, patient response lasted for approximately 5.5 months. Roughly 25% of patients had previously received anti–PD-L1/PD-1 therapy. Notably, patients who responded to erdafitinib treatment had not previously responded to anti–PD-L1/PD-1 therapy.

Adverse events frequently reported with erdafitinib use are increased phosphate level, mouth sores, feeling tired, change in kidney function, diarrhea, dry mouth, nails separating from the bed or poor formation of the nail, change in liver function, low sodium levels, decreased appetite, change in sense of taste, anemia, dry skin, dry eyes, and hair loss. Other adverse events include redness, swelling, hand foot syndrome, constipation, stomach pain, nausea, and muscle pain.—Hina Khaliq

Source: US Food and Drug Administration. FDA approves first targeted therapy for metastatic bladder cancer. April 12, 2019. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm635906.htm. Accessed April 12, 2019.