Sarepta Leapfrogs Pfizer In The US, Launching DMD Gene Therapy Pivotal Trial

Sarepta Therapeutics, Inc. appears to have a lead in the US over Pfizer Inc. in the race to bring the first gene therapy to market for Duchenne Muscular Dystrophy (DMD). Sarepta announced the initiation of the pivotal EMBARK trial on 4 October, testing its investigational gene therapy SRP-9001 in the US, Europe and Asia.

Sarepta and its ex-US partner Roche Holding AG have been in a close race with Pfizer, which has already started a Phase III trial, CIFFREO, testing the gene therapy fordadistrogene movaparvovec in some countries – but the trial was delayed in the US. Pfizer updated investors in May on the status of the program and warned the delay due to certain technical questions from the US Food and Drug Administration would extend into the second half of the year