Johnson & Johnson has announced that the US Food and Drug Administration (FDA) has granted full approval for BALVERSA® (erdafitinib) in the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) possessing susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations. 

This approval is specifically for patients whose disease has progressed after at least one prior systemic therapy, excluding those eligible for and not having received prior PD-1 or PD-L1 inhibitor therapy.

The FDA's decision follows the conversion of BALVERSA®'s accelerated approval in April 2019 to a full approval, based on the clinical and overall survival benefits observed in the Phase 3 THOR study. BALVERSA® stands out as the first oral FGFR kinase inhibitor to receive approval and serves as the initial targeted treatment for patients with mUC and FGFR alterations.

Approximately 20 percent of mUC patients exhibit FGFR3 genetic alterations, facing a challenging prognosis after one or more lines of systemic therapy, including checkpoint inhibitors. The Phase 3 THOR study (NCT03390504) demonstrated a 36 percent reduction in the risk of death with BALVERSA® compared to chemotherapy in the second-line setting for patients previously treated with a PD-1 or PD-(L)1 inhibitor. 

Those in the BALVERSA® arm lived a median of over four months longer, marking a significant advancement in the treatment landscape (Hazard Ratio (HR) 0.64; [95 percent Confidence Interval (CI), 0.47-0.88]; p=0.0050).

Kiran Patel, MD, Vice President, Clinical Development, Solid Tumors, Johnson & Johnson Innovative Medicine, expressed optimism about BALVERSA®'s continued promise in targeted therapy for advanced bladder cancer based on Phase 3 data and stated, "This important milestone reinforces our commitment to advance innovative, precision therapies in oncology and confirm the role of targeted therapy in the treatment of bladder cancer."

The US Prescribing Information for BALVERSA® includes warnings and precautions related to ocular disorders, hyperphosphatemia, and embryo-fetal toxicity. Common adverse reactions, including laboratory abnormalities, comprise increased phosphate, nail disorders, stomatitis, diarrhea, and others.

Johnson & Johnson facilitates access to BALVERSA® and related patient services through a single-source specialty pharmacy provider, US Bioservices, aligning with the company's ongoing commitment to providing high-quality products, services, access, and support to healthcare professionals and patients.