The FDA granted an accelerated approval to erdafitinib (Balversa) for the treatment of patients with locally advanced or metastatic bladder cancer with FGFR3 or FGFR2 genetic mutations who had progressed on previous platinum-based chemotherapy.
This FDA action marks the first approval of a targeted agent for the treatment of advanced bladder cancer. “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, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a 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 agency based its action on a clinical trial that included 87 patients with locally advanced or metastatic bladder cancer with the specified mutations and who progressed on or following chemotherapy. The overall response rate was 32.2%, including 2.3% of patients who experienced a complete response and almost 30% with a partial response. The average response time was 5.5 months.
About 25% of patients were previously treated with PD-L1/PD-1 therapy, which is standard for patients who have locally advanced or metastatic bladder cancer, from which some of these patients responded to erdafitinib after they failed to respond to anti PD-L1/PD-1 therapy.
Common adverse events (AEs) from erdafitinib included increased phosphate level, mouth sores, fatigue, 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 AEs were redness, hand foot syndrome, constipation, stomach pain, nausea, and muscle pain.
Practitioners should also be aware that erdafitinib may cause serious eye problems. If the patient experiences any symptoms in their eyes, they should notify their healthcare professionals immediately. Doctors and nurses should also closely monitor patients’ blood phosphate levels, and discuss proper contraception for both males and females who are of child-bearing age, as the drug may be harmful to a developing fetus or a newborn baby.