FDA Okays First Targeted Drug for Metastatic Bladder Cancer

Nick Mulcahy


April 12, 2019

The US Food and Drug Administration (FDA) granted accelerated approval to erdafitinib (Balversa, Janssen) for the treatment of locally advanced or metastatic bladder cancer with genetic alterations known as fibroblast growth factor (FGFR) 3 or 2.

Erdafitinib is indicated for use in patients who have first progressed on platinum-containing chemotherapy. Also, patients must be selected for therapy with a newly approved companion diagnostic device, the therascreen FGFR RGQ RT-PCR Kit (Qiagen Manchester).

"Today's approval represents the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer," said Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.

He explained that FGFRs regulate important biological processes including cell growth and division during development and tissue repair.

"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," added Pazdur.

The FDA observed that bladder cancers are associated with genetic mutations that are present in the patient's bladder or entire urothelium (lining of the lower urinary tract). FGFR alterations are present in approximately one in five patients with recurrent and refractory bladder cancer.

The efficacy and safety of erdafitinib were established in a clinical trial that included 87 patients with locally advanced or metastatic bladder cancer and FGFR3 or FGFR2 genetic alterations that had progressed following treatment with chemotherapy.

The overall response rate was 32.2%, with 2.3% having a complete response and nearly 30% having a partial response. The average duration of response was about 5.5 months. Roughly a quarter of the patients were previously treated with anti-PD-L1/PD-1 therapy, a standard treatment for patients with locally advanced and metastatic bladder cancer. Notably, responses to erdafitinib occurred in patients who were nonresponders to anti-PD-L1/PD-1 therapy.

Further clinical trials are required to confirm erdafitinib's clinical benefit.

Common side effects with erdafitinib included increased phosphate level, mouth sores, fatigue, change in kidney function, diarrhea, dry mouth, onycholysis, change in liver function, low sodium levels, decreased appetite, change in sense of taste, anemia, dry skin, dry eyes, and hair loss. Other side effects include redness, swelling, hand-foot syndrome, constipation, stomach pain, nausea, and muscle pain.

Erdafitinib may cause serious eye problems, including inflamed eyes, inflamed cornea, and disorders of the retina. Patients are advised to have eye examinations intermittently and to tell their healthcare professional right away if they develop blurred vision, loss of vision, or other visual changes.

Clinicians are advised to check blood phosphate levels between 14 and 21 days after starting treatment and monthly, and to increase the dose of erdafitinib in patients whose serum phosphate is below target level.

Erdafitinib may be teratogenic. Clinicians should advise male and female patients and their partners accordingly.

Erdafitinib must be dispensed with a patient medication guide that describes important information about the drug's uses and risks.

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