FDA Approves First Targeted Therapy to Treat Metastatic Bladder Cancer

The Food and Drug Administration (FDA) granted accelerated approval to Balversa (erdafitinib) to treat locally advanced or metastatic bladder cancer with a type of susceptible genetic alterations known as FGFR3 or FGFR2, in patients who have progressed during or following prior platinum-containing chemotherapy.

This marks the first approved targeted therapy for this patient population. “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 alterations 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.

Bladder cancer, the sixth most common cancer in the United States, can be associated with genetic mutations that are present in the patient's bladder or entire urothelium (the lining of the lower urinary tract). Fibroblast growth factor (FGFR) alterations are present in approximately one in five patients with recurrent and refractory bladder cancer.

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

The FDA based its approval on data from the multicenter, open-label, single-arm Study BLC2001 trial of 87 patients with 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. Balversa was administered until disease progression or unacceptable toxicity.

The overall response rate among those treated with Balversa was 32.3%, including 2.3% with a complete response and almost 30% with a partial response.

According to an FDA press release, common side effects among patients in the pivotal trial included 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 salt (sodium) levels, decreased appetite, change in sense of taste, low red blood cells (anemia), dry skin, dry eyes and hair loss.