Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals living with Duchenne amenable to skipping exons 45 or 53, respectively.
Both therapies are currently approved under the FDA’s accelerated approval pathway, which allows earlier access based on surrogate endpoints while requiring confirmatory evidence of clinical benefit. Following the initial readout from the Phase 3 ESSENCE trial, Sarepta is required to submit additional data to support a potential conversion from accelerated to traditional approval.
According to Sarepta, the company requested a meeting with FDA to discuss submitting sNDAs and has since received feedback from the agency confirming that Sarepta can move forward with the submissions. The company indicated that they intend to submit the sNDAs, including data from the ESSENCE confirmatory study as well as real-world evidence, by the end of April. Once received, the FDA will review the sNDAs as part of its determination on whether the therapies meet the standard for traditional approval. As part of this process, the agency may grant traditional approval or determine that more data is required.
PPMD is grateful to the families who participated in AMONDYS 45 and VYONDYS 53 studies, and to the investigators who supported them. We commend the FDA for their commitment to innovation with appropriate regulatory flexibility and appreciate Sarepta’s efforts to submit a more robust data package. We will continue to share updates as information becomes available.
Read Sarepta’s press release here.
Read Sarepta’s community letter:
March 19, 2026
Dear Duchenne Community,
We are writing to share an important update that reflects continued progress in the journey we are all on together.
Today, Sarepta provided an update on its interactions with FDA regarding AMONDYS 45 and VYONDYS 53 following completion of ESSENCE, our post-marketing requirement study that was completed last year.
What guidance did we get from FDA on next steps for AMONDYS and VYONDYS?
Following the completion of ESSENCE, Sarepta sought guidance from FDA
on submitting supplemental new drug applications (sNDAs) to convert the accelerated FDA approvals of AMONDYS 45 and VYONDYS 53 to traditional approvals.Today we shared that FDA provided feedback and we plan to submit data from ESSENCE and additional published real-world evidence as part of the sNDAs packages to support the review.
What happens next?
Sarepta plans to submit supplemental regulatory applications by the end of April seeking traditional approval for AMONDYS 45 and VYONDYS 53. The regulatory applications will include the data from ESSENCE along with an extensive body of real-world evidence supporting the impact of these therapies on Duchenne.
Dystrophin restoration therapies aim to preserve and protect remaining muscle and the body of data over the last decade continues to grow.
For families, patients, caregivers, clinicians, and advocates living with Duchenne muscular dystrophy, progress is rarely measured in headlines. It is measured in everyday moments— steps taken, strength preserved, time gained, hope sustained over years, not months.
What stands out is the continued commitment to listen, to learn, and to bring forward all available evidence—clinical data alongside real-world outcomes—to ensure that decisions reflect the full picture of life with Duchenne.
To the families who have contributed to research, trials, and care—your strength continues to shape the path forward. Your participation, your advocacy, and your perseverance are the foundation of every advancement we see today. Thank you.
Please reach out to advocacy@sarepta.com if you have any questions.
Wendy
Wendy Erler
Senior Vice President, Patient Affairs
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