FDA Approves First Targeted Therapy for Bladder Cancer

The US Food and Drug Administration (FDA) has approved Balversa (erdafitinib) as the first targeted therapy for bladder cancer.  The drug is for adults with advanced bladder cancer with a mutation in the FGFR3 or FGFR2 gene, and that has gotten worse after treatment with chemotherapy. The FDA also approved a test to look for these gene mutations.

FGFRs (fibroblast growth factor receptors) are proteins on cells that help them grow and divide. Some people with bladder cancer have changes in the genes that tell the cells to make FGFRs, which can help the cancer cells grow. Balversa works by targeting cells with these genetic changes.

The FDA based its approval on a clinical trial of 87 people with advanced bladder cancer that had FGFR3 or FGFR2 mutations, and that got worse after chemotherapy. The overall response rate – the proportion of people whose tumors shrank during the trial – was 32.2%, or about ⅓. Most of them had a partial response, which means the cancer shrank but didn’t go away completely. The improvements lasted an average of 5 ½ months. Two people had a complete response, which means their cancer could no longer be detected after the treatment.

The FDA evaluated Balversa using its accelerated approval program, for drugs that show promise against a serious disease. The company that makes the drug must continue to submit clinical information after approval, to demonstrate its benefit. The FDA also granted Balversa breakthrough therapy status, another approach designed to speed up the availability of drugs to treat serious diseases.

The most common side effects of Balversa were increased phosphate levels (which can weaken bones), low sodium levels, mouth sores, tiredness, kidney or liver problems, diarrhea, dry mouth, nail problems, decreased appetite, taste changes, low red blood cell counts, dry skin, dry eyes, and hair loss.

The FDA advises people taking Balversa to have regular eye exams because Balversa can cause serious eye problems, too. Patients should also have their blood phosphate level checked regularly.

Women should not take Balversa while pregnant or breastfeeding. Men should not father a child while taking Balversa.

Balversa is marketed by Janssen Pharmaceutical, and the companion test, therascreen FGFR RGQ RT-PCR Kit is marketed by QIAGEN Manchester, Ltd.

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