Sarepta secures early 2027 FDA decision for Duchenne drugs, pushing past confirmatory fail

 

Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial.

The FDA has formally accepted Sarepta Therapeutics’ application for the full approval of its two Duchenne muscular dystrophy medicines, with a verdict expected on or before Feb. 28, 2027.

Sarepta is looking to convert the accelerated approval for Amondys 45 and Vyondys 53—exon-skipping therapies given the greenlight in 2021 and 2019, respectively—into full approval. In November last year, results from the confirmatory Phase 3 ESSENCE trial showed that neither agent elicited significant motor function improvements versus placebo.

Still, analysts at Oppenheimer view the FDA’s acceptance of Sarepta’s applications as a “positive signal,” especially because Sarepta on Tuesday said the submission also includes supplemental “substantial published real-world evidence and the favorable and consistent safety profiles of both exon-skipping therapies.”

“We think inclusion of positive real-world safety data improves likelihood of conversion to full approval,” the analysts told investors in a note on Tuesday, noting that “safety will be key” for the FDA’s verdict.

Jefferies shared this sentiment, saying in its own Tuesday note that real-world data suggest the exon-skippers can “help patients remain ambulatory, off ventilators, and out of ERs/hospitals.” Given that side effects are mostly mild or moderate, “the overall benefit/risk profile remains favorable” for Amondys and Vyondys, the analysts added.

Beyond the beefed-up submission, Jefferies also pointed to other broader changes that could boost Sarepta’s chances of an approval. For one, the firm noted the exit of former Commissioner Marty Makary—as well as that of Vinay Prasad, ex-director of the Center for Biologics Evaluation and Research—which in turn could give way to a regulator that is more lenient toward rare diseases.

“We are closely monitoring the new FDA’s stance toward rare diseases,” Jefferies wrote. The firm also noted some “softening” on the regulator’s part regarding recent rejections, “alongside cases where the agency has revised its view on supportive data.”

Replimune, which owns the twice-rejected melanoma therapy RP1, said last week that the FDA has accepted a resubmission after some “collaborative dialogue” with the company. A decision is expected by Aug. 2, with an advisory committee meeting planned.

Similarly, uniQure now plans a Q3 submission for its Huntington’s disease gene therapy after a 180-degree-turn from the FDA, which had previously wanted a sham surgery–controled trial to establish the benefits of the investigational treatment—but has now instead agreed to the use of uniQure’s externally controlled Phase 1/2 trial.

Full approvals for Amondys and Vyondys would hand Sarepta a much-needed win after the biotech last year was rocked by patient deaths associated with its gene therapies. Its FDA-approved Duchenne gene therapy Elevidys was linked to two deaths—one in March 2025 and another in June—followed by a third mortality in a patient treated with an investigational gene therapy for limb-girdle muscular dystrophy.

Following this episode, the biotech pivoted away from gene therapies to instead focus on RNA interference modalities.

https://www.biospace.com/fda/sarepta-secures-early-2027-fda-decision-for-duchenne-drugs-pushing-past-confirmatory-fail