The Food and Drug Administration (FDA) has granted accelerated approval to Balversa (erdafitinib; Janssen) for the treatment of adults with locally advanced or metastatic urothelial carcinoma with susceptible fibroblast growth factor receptor (FGFR)3 or FGFR2 genetic alterations, and who have progressed during or following ≥1 line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Balversa, a first-in-class oral FGFR kinase inhibitor, works by inhibiting FGFR phosphorylation and signaling. It also decreases cell viability in cell lines expressing FGFR genetic alterations, including point mutations, amplifications, and fusions.

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The approval was supported by data from a phase 2 multicenter, open-label, single-arm trial (N=87) of patients with locally advanced or metastatic bladder cancer with FGFR3 or FGFR2 genetic alterations that had progressed following treatment with chemotherapy. The data showed an overall response rate (ORR) of 32.2% (95% CI, 22.4, 42.0) with a complete response seen in 2.3% of patients and a partial response observed in 29.9% of patients. The median duration of response was 5.4 months (95% CI, 4.2, 6.9) in Balversa-treated patients.

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Concurrently, the FDA has also approved the Qiagen therascreen FGFR RGQ Reverse-transcription (RT)-polymerase chain reaction (PCR) Kit for use as a companion diagnostic with Balversa for this indication.

The most common adverse reactions reported with Balversa include increased phosphate, stomatitis, fatigue, increased creatinine, diarrhea, dry mouth, onycholysis, increased alanine aminotransferase and alkaline phosphatase, decreased sodium, decreased appetite, decreased albumin, dysgeusia, decreased hemoglobin, dry skin, increased aspartate aminotransferase, decreased magnesium, dry eye, alopecia, palmarplantar erythrodysesthesia syndrome, constipation, decreased phosphate, abdominal pain, increased calcium, nausea, and musculoskeletal pain.

“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. “Today’s approval represents the first personalized treatment targeting susceptible FGFR genetic alternations for patients with metastatic bladder cancer.” Balversa will be supplied in 3mg, 4mg, and 5mg strength tablets.

Balversa will be supplied in 3mg, 4mg, and 5mg strength tablets.

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This article originally appeared on MPR