September 17, 2021

FDA Approves First Targeted Therapy for Metastatic Bladder Cancer

By SPT Staff
Published Online: Thursday, Apr 18, 2019

Officials with the FDA granted accelerated approval to erdafitinib (Balversa, Janssen) for adult patients with locally advanced or metastatic bladder cancer. This treatment is used for treating the indicated cancer that has a type of susceptible genetic alteration­­, FGFR3 or FGFR2, and that has progressed during or following prior platinum-containing chemotherapy.

Erdafitinib was granted a Breakthrough Therapy designation, and it received Accelerated Approval from the FDA. The Accelerated Approval designation enables the agency to focus on drugs for serious conditions that fill an unmet medical need, using clinical trial data that is thought to predict a clinical benefit to patients. Further clinical trials are required to confirm the drug’s clinical benefit.

The FDA also approved the therascreen FGFR RGQ RT-PCR Kit (Qiagen) for use as a companion diagnostic with erdafitinib for this therapeutic indication. According to the agency, patients should be selected for therapy with erdafitinib using this 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 alterations for patients with metastatic bladder cancer,” said Richard Pazdur, MD, 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 prepared 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.”

The most common type of bladder cancer is transitional cell carcinoma, also called urothelial carcinoma. Bladder cancers are associated with genetic mutations that are present in the patient's bladder or entire the lining of the lower urinary tract. Bladder cancer is the sixth most common cancer in the United States. Fibroblast growth factor (FGFR) alterations are present in approximately 1 in 5 patients with recurrent and refractory bladder cancer.

The efficacy of erdafitinib was studied in a clinical trial that included 87 patients with locally advanced or metastatic bladder cancer, with FGFR3 or FGFR2 genetic alterations, that had progressed following treatment with chemotherapy. The overall response rate in these patients was 32.2%, with 2.3% having a complete response and nearly 30% having a partial response. The response lasted for an average of approximately 5.5 months.

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