Cambridge, Mass. — Sarepta Therapeutics said the U.S. Food and Drug Administration has accepted supplemental New Drug Applications for AMONDYS 45 and VYONDYS 53 for the treatment of Duchenne muscular dystrophy.
The FDA set a target action date of Feb. 28, 2027, under the Prescription Drug User Fee Act.
The applications seek to convert the accelerated approvals of AMONDYS 45, or casimersen, and VYONDYS 53, or golodirsen, into traditional approvals.
Sarepta said the submissions are supported by results from the ESSENCE confirmatory study, 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.”
AMONDYS 45 is approved for patients with Duchenne muscular dystrophy who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. VYONDYS 53 is approved for patients with a confirmed mutation amenable to exon 53 skipping.
“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.”
