Vesleteplirsen

Sarepta Therapeutics filed for accelerated approval of SRP-9001, a gene therapy for Duchenne muscular dystrophy (DMD), aiming for a 2023 launch. The therapy, developed with Roche, targets ambulatory DMD patients, using an AAV vector to deliver a dystrophin gene. Approval is sought based on early-stage studies, awaiting phase 3 EMBARK results. Sarepta also markets three exon-skipping DMD drugs, with SRP-9001 offering a one-dose solution. Roche holds exclusive rights outside the US.

Sarepta cancels Vesleteplirsen program due to updated risk-benefit analysis, FDA feedback, and evolving Duchenne therapeutic landscape. The company focuses on RNA-based therapies, including exon-skipping ASOs and gene therapies, for Duchenne and other neurological conditions. Despite achieving FDA approval for Elevidys, Sarepta prioritizes safety over development, discontinuing SRP-5051 after side effects emerged in trials.

Sarep­ta Ther­a­peu­tics is stop­ping de­vel­op­ment of vesleteplirsen, an ex­per­i­men­tal ex­on 51-skip­ping ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy pa­tients with cer­tain mu­ta­tions.

Sarepta Therapeutics has halted development of vesleteplirsen, a more potent version of its DMD therapy Exondys 51, citing risk-benefit considerations, FDA feedback, and the evolving DMD therapeutic landscape. Elevidys, Sarepta's gene therapy, is rapidly gaining ground, with $181 million in Q3 sales compared to $249 million for Exondys 51 and its counterparts.

Sarepta Therapeutics discontinues vesleteplirsen development for Duchenne muscular dystrophy due to FDA feedback and risk-benefit analysis, focusing on Elevidys gene therapy which generated $190.5 million in Q3 2024.