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

[March 08, 2023]  (Reuters) - The U.S. Food and Drug Administration (FDA) 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.

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

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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