FDA Approves Erdafitinib, First Targeted Therapy for Metastatic Bladder Cancer

The FDA has approved the first targeted therapy for bladder cancer with its accelerated approval of erdafitinib (Balversa) for the treatment of adult patients with locally advanced or metastatic bladder cancer with fibroblast growth factor receptor (FGFR)3 or FGFR2 mutations who have progressed on platinum-containing chemotherapy.

FGFR mutations account for approximately 1 in 5 patients with recurrent or refractory bladder cancer. The drug was approved alongside QIAGEN’s therascreen FGFR RGQ RT-PCR Kit as a companion diagnostic to select which patients will most likely benefit from the therapy.

“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,” 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 statement. “Today’s approval represents the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer.”

Erdafinitib’s efficacy was demonstrated in a phase 2 clinical trial of 87 patients with locally advanced or metastatic disease that had progressed following or during treatment with chemotherapy. Patients either had FGFR3 mutations or FGFR gene fusions.

Treatment with the FGFR kinase inhibitor resulted in an overall response rate of 32.2%, with 2.3% of these patients having a complete response and nearly 30% having a partial response. These responses held for a median duration of 5.4 months. Responders included those who had not responded to programmed cell death protein 1 or programmed death ligand-1 therapy. There were no confirmed responses among the 6 patients with FGFR2 fusions.

“We recognize the significant unmet need that persists in the treatment of men and women diagnosed with this form of urothelial carcinoma, and we have worked expeditiously to develop Balversa for patients in close consultation with the FDA,” Peter Lebowitz, MD, PhD, global therapeutic area head, oncology, Janssen Research & Development, said in a statement. “We look forward to the continued development of Balversa to understand how this important new therapy may further inform the care of patients with metastatic urothelial carcinoma and its investigational use in other cancers where FGFR alterations may be present in the future.”

Common side effects included increased phosphate level, mouth sores, feeling tired, change in kidney function, low sodium levels, diarrhea, dry mouth and change in liver function. Other side effects included redness, swelling, peeling, or tenderness on the hands or feet.