Sarepta shares PhII data for next-gen exon skipping drug, but regulatory pathway is unclear

Sarepta Therapeutics has released Phase II data for its next-generation exon-skipping drug, but the regulatory pathway for this therapy is unclear given the context provided in the search results.

1. Sarepta received a favorable recommendation from the FDA Advisory Committee for the approval of their gene therapy for Duchenne muscular dystrophy (DMD) called Sarepta's gene therapy 'Elevidys' micro-dystrophin. The FDA granted accelerated approval to this therapy based on the data that showed an increase in micro-dystrophin expression.

2. Sarepta is seeking approval for the therapy with the plan to submit a Biologics License Application (BLA) supported by biomarker data. However, the FDA is reportedly unconvinced of the efficacy measured only by micro-dystrophin levels, which may not directly correlate with functional improvements.

3. The regulatory pathway for Sarepta's next-generation exon-skipping drug cannot be conclusively determined given the information available. The search results focus primarily on Sarepta's gene therapy for DMD and do not directly address their other exon-skipping drug candidates' regulatory prospects.

4. The Phase III trial (EMERGENE) for a different gene therapy, SRP-9003, which is for limb-girdle muscular dystrophy Type 2E, has started subject screening. SRP-9003 uses the AAVrh74 vector for targeted delivery, which may have implications for other gene therapies the company is developing, but it does not directly inform the regulatory pathway for Sarepta's next-generation exon-skipping drug.

In summary, while Sarepta's gene therapy for DMD has received a favorable review from an FDA Advisory Committee and was granted accelerated approval, the regulatory pathway for their next-generation exon-skipping drug is unclear, and the provided search results do not conclusively address this specific topic.