US FDA to review Sarepta’s Duchenne gene therapy for traditional approval
ReutersUS FDA to review Sarepta’s Duchenne gene therapy for traditional approval
Feb 16 (Reuters) – Sarepta Therapeutics (SRPT.O) said on Friday that the U.S. Food and Drug Administration 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 14.5% in premarket 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.
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.
When debating the accelerated approval in May last year, some of the 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 all secondary study goals with statistically significant results, and no new safety signals were observed.
The FDA’s decision to not hold an advisory panel meeting, and its priority review despite the trial failure comes in the backdrop of the regulator looking at ways to boost commercialization of gene and cell therapies for patients with rare diseases.
Besides the traditional approval, Sarepta’s application also seeks the removal of 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
Source: Reuters
