Drugs

Sarepta to Submit Amondys 45 and Vyondys 53 sNDAs

Sarepta says it will submit to FDA by the end of April sNDAs to convert the accelerated approvals of its Duchenne muscular dystrophy drugs Amondys 45 (casimersen) and Vyondys 53 (golodirsen) to traditional approvals. A company statement says the decision to submit the sNDAs follows a meeting with FDA on converting the approvals.

Sarepta says its request is supported by data from the ESSENCE confirmatory study, substantial published real-world evidence supporting treatment, and the favorable safety profiles of both therapies. "Topline results from ESSENCE presented at 2026 Muscular Dystrophy Association Clinical & Scientific Congress found that numerical trends favored treatment versus placebo; however, the observed difference of 0.06 steps/second in least square means (LSM), did not reach statistical significance (P=0.309) on the primary endpoint, the 4-step ascend velocity at 96 weeks," it notes.

“Sarepta has received feedback from the agency confirming that we can submit our data from ESSENCE and real-world evidence as part of the sNDAs,” the statement says. “The adequacy of the data to support conversion to traditional approval will be a matter of review.”

Amondys 45 and Vyondys 53 are described as exon-skipping therapies approved under FDA’s accelerated approval pathway for patients with Duchenne muscular dystrophy who have mutations amenable to exon 45 and exon 53 skipping, respectively.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome