STAT+: Wagering on FDA changes, Regenxbio will submit Duchenne gene therapy for approval

Regenxbio announced on Wednesday that it will seek accelerated approval for its gene therapy targeting Duchenne muscular dystrophy. This decision follows a statement made by the company just one month prior, indicating that regulatory bodies required an additional trial and that an application would be delayed. The shift in strategy coincides with apparent changes in the Food and Drug Administration's (FDA) stance on several previously rejected or deferred drug applications. Regenxbio also revealed on Monday that the FDA has agreed to re-evaluate a gene therapy for Hunter syndrome, which had been rejected only four months earlier. Notably, Marty Makary and Vinay Prasad, who were associated with the FDA during that prior rejection, have recently departed the agency. The company's press release on Wednesday did not specify if regulatory reviewers had altered their position on the Duchenne gene therapy.