FDA Approves Janssen's Balversa (Erdafitinib) as First Targeted Therapy for Metastatic Bladder Cancer with FGFR Alterations

Last Friday, the FDA approved Johnson & Johnson’s innovative small-molecule drug Balversa (erdafitinib) for the treatment of adult patients with locally advanced or metastatic bladder cancer who harbor susceptible genetic alterations in FGFR3 or FGFR2 and whose disease has progressed before or after platinum-containing chemotherapy.

Balversa was granted Breakthrough Therapy Designation, alongside the approval of a companion diagnostic device developed by Qiagen for detecting FGFR2 and FGFR3 mutations. This device will be used to identify patients with FGFR2 and FGFR3 mutations who are eligible for Balversa treatment.

“We are in an era of more personalized or precision medicine, where the ability to target cancer treatments to a patient’s specific genetic mutations or biomarkers is becoming standard and making progress in new disease types. Today’s approval represents the first personalized therapy targeting susceptible FGFR gene alterations in patients with metastatic bladder cancer,” said Dr. Richard Pazdur, 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. “FGFR regulates key processes involved in cell growth, division, development, and tissue repair. The drug works by targeting specific genetic mutations in FGFR.”

Bladder cancer is the sixth most common cancer in the United States. The most common type of bladder cancer is urothelial carcinoma. Bladder cancer is associated with genetic mutations present in the patient’s bladder or urothelium (the lining of the lower urinary tract). Approximately one-fifth of patients with recurrent and refractory bladder cancer harbor fibroblast growth factor receptor (FGFR) alterations. In recent years, immunotherapies such as Merck’s Keytruda and Roche’s Tecentriq have become standard treatments for metastatic bladder cancer.

Molecular Formula of Balversa (Erdafitinib)

Balversa’s approval was based on its performance in a clinical trial involving 87 patients. These patients harbored specific FGFR2 or FGFR3 genetic alterations. The overall response rate was 32.2%, with 2.3% achieving complete responses and nearly 30% achieving partial responses. The median duration of response was approximately five and a half months. About one-quarter of the patients in the study had previously received PD-L1/PD-1 inhibitor therapy, which is the standard treatment for patients with locally advanced or metastatic bladder cancer.

Balversa is also effective in patients who have previously not responded to PD-L1/PD-1 therapy. Treatment is also associated with significant side effects, including the risk of serious ocular problems such as keratitis and retinal disorders. The FDA recommends that patients undergo periodic ophthalmologic examinations.