Regenxbio reports successful Duchenne gene therapy trial, clearing path to FDA filing
read at STAT ↗Positive readoutPositive
Regenxbio announced that its experimental gene therapy for Duchenne muscular dystrophy met its primary endpoints in a recent clinical trial, producing sufficient levels of a truncated muscle protein.
The results are positioned as a potential improvement over Sarepta Therapeutics' Elevidys, which has been shadowed by safety concerns after two recipients died from liver failure.
CEO Curran Simpson said the data satisfy all requirements for an accelerated approval filing, and the company intends to submit the data to the FDA in the near future.
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This writeup was produced by pharmadog from original reporting by STAT.
Original headline: “STAT+: Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission”
read at STAT ↗543 words · retrieved May 14
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