FDA Extends Review of BioMarin's Bleeding Disorder Therapy

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FDA extends review of BioMarin's bleeding disorder therapy. medtwitter FDA

- The U.S. Food and Drug Administration has extended the review of BioMarin Pharmaceutical Inc's experimental gene therapy for adults with a severe bleeding disorder, the latest delay in the company's quest to gain approval in the country.

The FDA said late on Monday it needed more time to review a three-year analysis from the company's ongoing late-stage study, which BioMarin submitted earlier this year, and will make a decision by June 30. The regulator had earlier set March 31 as its decision date. The potential one-time treatment, if approved, will be the first gene therapy for hemophilia A, a genetic bleeding disorder that affects one in 10,000 people and requires regular infusions of a blood clotting protein called factor VIII.

The FDA had rejected the drug in August 2020, citing the need for longer-term data. It was granted conditional marketing authorization in the European Union in August last year.

 

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