U.S. FDA extends review of BioMarin's bleeding disorder therapy

Signage is seen outside of FDA headquarters in White Oak, Maryland
Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly

March 7 (Reuters) - The U.S. Food and Drug Administration (FDA) has extended the review of BioMarin Pharmaceutical Inc's (BMRN.O) 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.

Reporting by Mariam Sunny in Bengaluru Editing by Vinay Dwivedi

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