Aiming for fourth nod, Sarepta files another DMD gene therapy to FDA; Axsome headed toward migraine resubmission

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

Sarepta is asking for a conditional greenlight for delandistrogene moxeparvovec, also known as SRP-9001, based on a surrogate endpoint that the drugmaker thinks should translate into clinical benefit once borne out in a confirmatory study. That trial, dubbed EMBARK, is already fully enrolled, the biotech said.

After getting three antisense oligonucleotide therapies across the finish line, Sarepta is looking for a long-term treatment via gene therapy, and the approval request comes in light of two recent FDA blessings for gene…
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