ARTICLE | Regulation
Plus: June PDUFA date for Daiichi-Merck ADC, and updates from Lyndra, ImpriMed, Mundipharma and Novamab
By BioCentury Staff
December 23, 2023 1:40 AM UTC
Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it submitted an efficacy supplement and postmarketing data to FDA for Duchenne muscular dystrophy gene therapy Elevidys delandistrogene moxeparvovec, aiming to expand the drug’s label to all patients with a confirmed mutation in the DMD gene, regardless of age or ambulatory status, and to convert the drug’s accelerated approval to full approval. The company also requested priority review.
In June, FDA approved Elevidys in patients four through five years of age, making it the first gene therapy approved for the indication. In October, the company presented mixed results from the Phase III EMBARK study, in which Elevidys missed the primary endpoint of improvement in the North Star Ambulatory Assessment (NSAA) at 52 weeks versus placebo, but met four of five secondary endpoints, including the two key secondary endpoints: the time it takes the patient to rise from the floor and the time it take them to walk 10 meters…
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- Source: https://www.biocentury.com/article/651023/dec-22-quick-takes-sarepta-seeking-expanded-label-full-approval-of-dmd-gene-therapy