
FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy
James Spargo | May 11, 2023 | News story | Medical Communications | Duchenne Muscular Dystrophy, FDA, Musculo-skeletal disorder, Sarepta Therapeutics, gene therapy
The US Food and Drug Administration (FDA) has released a report in preparation for its 12 May decision on Sarepta Therapeutics’ gene therapy for the treatment of Duchenne muscular dystrophy (DMD).
SRP-9001 (delandistrogene moxeparvovec) is an adeno-associated virus (AAV) vector-based gene therapy which is designed to treat the proximate cause of DMD by replacing dysfunctional or missing dystrophin protein with a functional, shortened dystrophin, in cardiac, respiratory and skeletal muscles ‒ the key areas affected by DMD.
Sarepta submitted an FDA Biologics License Application (BLA) for SRP-9001 in September 2022.
However, in its documents the FDA cited a lack of “unambiguous evidence” that SRP-9001 is beneficial for patients with DMD. It also mentioned concerns around clinical trial evidence being translated into real-world benefits, safety issues and potential manufacturing problems in relation to the viral vectors used to deliver the treatment.
The FDA has until 29 May to decide whether or not to grant the gene therapy accelerated approval. If granted, Sarepta will be required to complete a confirmatory study.
James Spargo
Related Content

Rare diseases – spinal muscular atrophy
Spinal muscular atrophy (SMA) is a rare disease characterised by voluntary muscle weakness that can …

Five Facts about spinal muscular atrophy
1 Spinal muscular atrophy (SMA) is a genetic disease involving the loss of motor neurons …

MRM Health’s ulcerative colitis treatment receives FDA Investigational New Drug clearance
Microbial Resource Management (MRM) Health has announced that its lead programme, MH002, has received Investigational …





