Gene therapy granted FDA Priority Review

Dystrophin muscle protein

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy.  

The therapy, which is being developed in partnership with Roche, has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. 

Doug Ingram, President and Chief Executive Officer, Sarepta Therapeutics, said: “Duchenne is a relentlessly degenerative and invariably fatal disease, robbing children of muscle and function hourly and daily. Our BLA submission for an accelerated approval, along with the FDA’s acceptance of that BLA for filing and review, is a tremendously important milestone in our effort to bring a potentially life-changing gene therapy to Duchenne patients as rapidly as possible and we look forward to working with the FDA through the review process.” 

Duchenne is characterised by a mutation in the dystrophin gene that results in the lack of dystrophin, which acts as a shock absorber for muscle at the membrane. SRP-9001 is designed to treat the proximate cause of Duchenne by delivering to muscle a gene that codes for a shortened, functional form of dystrophin.  

In clinical results from more than 80 treated patients, SRP-9001 has demonstrated positive results at multiple time points, including one-, two- and up to four-years after treatment, in addition to demonstrating a consistent safety profile. 

It is currently being studied in the EMBARK (Study SRP-9001-301), a global, randomised, double-blind, placebo-controlled clinical trial, which has recruited 125 participants with Duchenne between the ages of four and seven. 

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