US FDA to review Sarepta's Duchenne gene therapy for traditional approval

[February 19, 2024]  (Reuters) - Sarepta Therapeutics said on Friday that the US FDA would review an application seeking traditional approval for its gene therapy to treat a muscle-wasting disorder by June 21, months after it failed the main goal of a confirmatory trial.

Shares of the company rose nearly 11% in morning trading. They briefly fell in October after data from the confirmatory study, but have recovered losses since then.

The company also said that the FDA did not plan to hold a meeting of its outside experts to discuss the new application.

William Blair analyst Tim Lugo said the lack of an advisory committee meeting is a positive sign. A traditional approval represents a $1 billion annual sales opportunity, he said.

The therapy, Elevidys, was granted accelerated approval in June last year to treat Duchenne muscular dystrophy, an inherited progressive muscle-wasting disorder that almost always affects young boys.

The initial approval was given for children aged between 4 and 5 years who can walk, based on a mid-stage trial, where the gene therapy produced a mini version of the dystrophin protein needed to help keep muscles intact.

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

When debating the accelerated approval in May last year, some panel members had said that Sarepta's late-stage trial to confirm the benefits of the gene therapy would be influential.

However, in October Elevidys failed the confirmatory trial's goal of significantly improving motor function in patients aged between 4 and 7 years.

Still, the therapy had met the secondary goals of significant improvement in the time it took for patients to rise from the floor and their ability to walk 10 meters.

Sarepta's application also seeks approval without restrictions on age and walking ability around the use of the gene therapy.

(Reporting by Christy Santhosh and Leroy Leo in Bengaluru; Editing by Shailesh Kuber)

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