Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®

About this update from Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45® (casimersen) and VYONDYS 53® (golodirsen) for the treatment of Duchenne muscular dystrophy (DMD). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action data of February 28, 2027. The sNDA submissions seek conversion of the accelerated approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals. The applications are supported by data from the ESSENCE confirmatory study, as well as substantial published real-world evidence and the favorable and consistent safety profiles of both exon-skipping therapies. “The FDA’s acceptance of these applications for review is an important step for the Duchenne community. The submissions draw on the ESSENCE study and years of published real-world evidence, which together offer a fuller understanding of how these therapies benefit patients and change the progression of disease,” said Louise Rodino‑Klapac, Ph.D., president of research & development and technical operations, Sarepta. “Within Duchenne, each amenable mutation defines an ultra-rare population—a small subset of an already rare disease. Across our exon skipping therapies, more than 1,800 people worldwide have been treated, and we continue to observe preservation of muscle function and slowed disease progression. In populations this small and in a disease where damage unfolds over years, real-world experience is essential to understanding how these therapies impact the disease course. We look forward to working with the FDA throughout the review.” “We appreciate the FDA’s continued willingness to apply regulatory adaptability in addressing the unique challenges of rare disease drug development. The acceptance of these supplemental applications for review reflects both the progress the Duchenne community has made over the past several years and the needs that remain, while maintaining a commitment to evaluating therapies with rigor,” said Pat Furlong, president and founder, Parent Project Muscular Dystrophy. “We are grateful for the FDA’s engagement with the Duchenne community and the Agency’s dedication to advancing therapeutic options through pathways adaptable to rare disease.” About S...

View stock analysis, news, and events for Sarepta Therapeutics, Inc.