FDA Approves Janssen’s Balversa, First Targeted Therapy for Metastatic Urothelial Carcinoma

FDA Approval stamp in red lettering

Janssen Pharmaceutical snagged accelerated approval for Balversa (erdafitinib) from the U.S. Food and Drug Administration (FDA). The green light marked the first targeted therapy approved for a type of bladder cancer in the United States.

Balversa, a once-daily, oral fibroblast growth factor receptor (FGFR) kinase inhibitor, was approved for the treatment of adults with locally advanced or metastatic urothelial carcinoma (mUC) which has susceptible fibroblast growth factor receptor (FGFR)3 or FGFR2 genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy. Balversa becomes the first FGFR kinase inhibitor approved by the FDA, Janssen said.

“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,” Richard Pazdur, director of the FDA’s Oncology Center of Excellence, said 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.”

Click to sign up for job alerts

Bladder cancer is the sixth most common cancer in the United States. The most common type of bladder cancer is transitional cell carcinoma, also called urothelial carcinoma. FGFR alterations are present in approximately one in five patients with recurrent and refractory bladder cancer, according to the FDA.

The FDA approval is based on positive Phase II data that demonstrated the efficacy and safety of Balversa for the treatment of adult patients with locally advanced or metastatic UC, whose tumors have certain FGFR alterations. The results demonstrated a 32.2 percent objective response rate, with 2.3 percent having a complete response and almost 30 percent having a partial response. Responders included patients who had previously not responded to treatment with checkpoint inhibitors. Results also showed a median duration of response of 5.4 months. When the study results were presented at the American Society of Clinical Oncology meeting last year, it was recognized as a "Best of ASCO" selection. The positive results from the Phase II trial helped earn Balversa Breakthrough Therapy Designation from the FDA.

Balversa can cause some irritation to the eyes, including inflamed eyes, inflamed cornea (and disorders of the retina. The FDA said patients on the medication are advised to have intermittent eye examinations while on the medication. Other side effects associated with the drug include increased phosphate level, mouth sores, feeling tired, change in kidney function, diarrhea and dry mouth.

Peter Lebowitz, global therapeutic area head of oncology at Janssen Research and Development, said they recognize there is a “significant unmet need that persists in the treatment of men and women diagnosed with this form of urothelial carcinoma.” Lebowitz said Janssen researchers have worked diligently to develop Balversa for these patients. Lebowitz added that Janssen will continue to develop the drug in order to understand how the first-of-its-kind therapy can “further inform the care of patients with metastatic urothelial carcinoma,” as well as in other cancer indications where FGFR alterations may be present.

In addition to the approval of Balversa, the FDA simultaneously approved a companion diagnostic for use with the newly approved drug called the Qiagen therascreen FGFR RGQ Reverse-transcription (RT)-polymerase chain reaction (PCR) Kit. The test detects the presence of FGFR alterations in the tumor tissue of patients with this form of cancer.

Back to news