FDA’s Accelerated Approval of Sarepta DMD Gene Therapy Comes Under Fire

The FDA’s authorization of therapies that fail their efficacy endpoints casts doubt on the regulator’s accelerated approval pathway, according to an article from David Rind, chief medical officer of the drug pricing non-profit Institute for Clinical and Economic Review, published Wednesday in the Journal of the American Medical Association.

In his viewpoint piece, Rind specifically called out Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec-rokl), which won the FDA’s accelerated approval in June 2023—despite falling short of its primary efficacy endpoint in two studies.