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Sarepta Therapeutics Says FDA Lifts Clinical Hold On SRP-5051 To Treat Duchenne Muscular Dystrophy
(RTTNews) - Genetic medicine maker Sarepta Therapeutics, Inc. (SRPT) announced Tuesday that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on SRP-5051 (vesleteplirsen), the Company's investigational, next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
After discussions with FDA and as part of the lift, Sarepta will adjust the global trial protocol to include expanded monitoring of urine biomarkers.
The hold in Part B of Study 5051-201, also known as MOMENTUM, followed a serious adverse event of hypomagnesemia. The Company provided the information to FDA to assess the adequacy of the risk mitigation and safety monitoring plan.
SRP-5051 is an investigational agent using Sarepta's PPMO chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.
MOMENTUM is a Phase 2, multi-arm, ascending dose trial of SRP-5051, infused monthly and will assess dystrophin protein levels in skeletal muscle tissue following SRP-5051 treatment.
The trial will enroll up to 60 participants, both ambulant and non-ambulant, between the ages of 7 to 21 at sites in the U.S., Canada, and the European Union.
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