Sarepta axes late-phase DMD drug over safety signal, FDA talks
Nov 7, 2024 · Sarepta kept going, ... The impact of the news extends beyond SRP-5051. Rodino-Klapac said Sarepta is discontinuing its entire PPMO franchise. PPMO is the chemistry platform behind SRP-5051 and ...
Sarepta's Elevidys gains 48% sequentially after FDA expansion
6 days ago · In the third quarter, Sarepta reported revenue from Elevidys at $181 million, routing analysts' consensus estimate of $160 million as it made a 48% sequential leap in sales. It was a significant ...
Sarepta Halts Development of Next-Gen DMD Drug, Reports
Nov 7, 2024 · On Wednesday’s earnings call, Sarepta’s head of R&D Louise Rodino-Klapac noted that these cases persisted even after discontinuing treatment, Endpoints News reported. The FDA informed Sarepta that the accelerated approval pathway “was not open” for vesleteplirsen based on current information on the drug, Rodino-Klapac added.
Sarepta scraps a Duchenne drug as gene therapy sales rise
Nov 7, 2024 · Sarepta Therapeutics is giving up on an experimental drug for Duchenne muscular dystrophy as sales rocket for its approved gene therapy for the disease. The decision to discontinue SRP-5051 was based on several factors, company executives told analysts on a conference call Wednesday. Safety concerns, feedback from the Food and Drug ...
Sarepta Announces Discontinuation of SRP-5051 Development for …
Nov 6, 2024 · Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and exon-skipping technology for individuals with Duchenne …
News & Press Releases - Sarepta Therapeutics
Read news and articles from Sarepta, a global biotechnology company developing potentially life-changing precision genetic medicine.
Sarepta Therapeutics Announces Expanded US FDA Approval of …
Jun 20, 2024 · CAMBRIDGE, Mass. -- (BUSINESS WIRE)--Jun. 20, 2024-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with …
Press Releases - Sarepta Therapeutics, Inc.
Sarepta Therapeutics Announces Recipients of LGMD Grant Awards for 2024; Program Supports Early Genetic Testing and Awareness
Sarepta Therapeutics Announces That U.S. FDA has Accepted for …
Nov 28, 2022 · Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta’s Gene Therapy for the Treatment of Ambulant Individuals with Duchenne Muscular Dystrophy. – Regulatory action date of May 29, 2023.
Muscular dystrophy patients get first gene therapy - NPR
Jun 22, 2023 · Sarepta based its request on how much micro-dystrophin it produces in patients' muscles — without direct evidence that's actually helping alleviate symptoms and prevent disease progression.
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