Home Medizin Die US-amerikanische FDA prüft die Duchenne-Gentherapie von Sarepta auf traditionelle Zulassung

Die US-amerikanische FDA prüft die Duchenne-Gentherapie von Sarepta auf traditionelle Zulassung

von NFI Redaktion

(Reuters) – Sarepta Therapeutics announced on Friday that the U.S. Food and Drug Administration (FDA) will review its application for traditional approval for their gene therapy to treat a muscle wasting disorder by June 21, months after missing the primary goal of a confirmatory study.

The company’s stock rose nearly 11% in morning trading. After data from the confirmatory study caused a brief dip in October, they have since recovered those losses.

The company also stated that the FDA does not plan to hold a meeting of its external experts to discuss the new application.

Tim Lugo, an analyst at William Blair, said that the absence of an advisory committee meeting is a positive sign. He also mentioned that traditional approval represents a $1 billion annual revenue opportunity.

The therapy, known as Elevidys, received accelerated approval in June of last year to treat Duchenne muscular dystrophy, an inherited progressive muscle-wasting disorder that almost exclusively affects young boys.

The initial approval was for children ages 4 to 5 who are able to walk, based on a mid-stage study where the gene therapy produced a mini version of the dystrophin protein needed to keep muscles intact.

During the debate over the accelerated approval in May of last year, some committee members stated that Sarepta’s late-stage study confirming the benefits of the gene therapy would be influential.

However, in October, Elevidys missed the target of the confirmatory study to significantly improve motor function in patients ages 4 to 7.

Nevertheless, the therapy did meet secondary goals of significantly improving the time it took for patients to rise from the floor and their ability to walk 10 meters.

Sarepta’s application also seeks approval for the use of the gene therapy without age or walking ability restrictions.

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

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