Regenxbio's DMD Gene Therapy Meets FDA Criteria
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- Regenxbio reported that its Duchenne muscular dystrophy gene therapy achieved high levels of a miniaturized muscle protein in a clinical trial.
- Regenxbio aims to develop a therapy that is more effective and safer than Sarepta Therapeutics’ Elevidys, which has faced safety concerns after two patient deaths from liver failure.
- Curran Simpson said the trial data meets all criteria for accelerated FDA approval, positioning the company for a regulatory filing.
Why it matters: Patients with Duchenne muscular dystrophy gain a promising, potentially safer therapy option, while Regenxbio prepares an FDA filing that directly competes with Sarepta’s Elevidys, potentially reshaping treatment standards for the disease.
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