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

Article excerpt

Regenxbio said Wednesday it will apply for accelerated approval for its gene therapy for Duchenne muscular dystrophy, even though regulators previously told the company they wanted it to first test the experimental drug in a new clinical trial. The announcement comes as the Food and Drug Administration seemingly reverses course on a slew of drugs it recently rejected or spurned. On Monday, Regenxbio said the FDA agreed to reconsider a Hunter syndrome gene therapy that it rejected just four months ago, when Marty Makary and Vinay Prasad were still at the agency’s helm. Both officials have left the FDA in recent weeks. Wednesday’s announcement is unusual, however. In a press release, the company gave no indication reviewers had changed their stance on the company’s Duchenne gene therapy.