Regenxbio Files Duchenne Gene Therapy, Reversing
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- Regenxbio said Wednesday it will apply for accelerated FDA approval for its Duchenne muscular dystrophy gene therapy, reversing a decision made just one month earlier to hold off on an application.
- Just last month, Regenxbio said regulators wanted to see another trial before it could proceed, prompting the company to pause its filing plans.
- The shift comes as the FDA appears to be reversing course on multiple previously rejected or spurned drugs, per the company's framing.
- On Monday, the FDA agreed to reconsider Regenxbio's Hunter syndrome gene therapy, which it had rejected four months earlier when Marty Makary and Vinay Prasad led the agency.
- Makary and Prasad, both recently departed from the FDA, were in charge when the Hunter syndrome therapy was rejected four months ago.
- Wednesday's Duchenne announcement is unusual because the company gave no indication that FDA reviewers had changed their stance on the Duchenne therapy specifically — making this a strategic wager rather than a confirmed regulatory opening.
Why it matters: Regenxbio is essentially betting the FDA's posture toward gene therapies has softened under post-Makary/Prasad leadership, submitting for Duchenne approval without any stated signal that reviewers changed their view. If the gamble pays off, Duchenne patients gain a potential accelerated-approval pathway; if not, the company has wasted capital and momentum on a filing likely to be rejected on the same grounds as a month ago.
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