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US FDA advisory committee votes on Sarepta SRP-9001 gene therapy to treat Duchenne muscular dystrophy ahead of a FDA’s final decision

19 May 2023

Last week an advisory committee for the US Food and Drug Administration (FDA) met to discuss Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy.

UPDATE: On 24 May 2023, Roche published an update in which they stated that the FDA’s final decision on accelerated approval of Sarepta Therapeutics’ SRP-9001 gene therapy for Duchenne muscular dystrophy has been postponed from 29 May 2023 to 22 June 2023. See Roche’s community letter for details.

The FDA advisory committee met on 12 May to discuss potentially accelerating approval of SRP-9001 (also known as delandistrogene moxeparvovec) gene therapy for the treatment of boys, who can still walk, with Duchenne muscular dystrophy (DMD). In a non-binding vote, the committee voted eight to six in favour of the accelerated approval of the gene therapy. A final decision will be made by the FDA by the end of the month, with consideration of the advisory committee vote.

While this doesn’t affect those with DMD in the UK or Europe, where the FDA does not have jurisdiction, the FDA’s decision might influence the next steps for the potential global approval of SRP-9001 in the future. Muscular Dystrophy UK has reached out to Roche for more information, and they have said that they will share as much detail as they can following the FDA’s final decision at the end of the month. We will share this information with our community as soon as we can.

Duchenne muscular dystrophy is a rare neuromuscular condition that mainly affects boys and men. People with DMD lack a protein called dystrophin, which is required to maintain the strength of muscles. SRP-9001 is a gene therapy for DMD developed by Sarepta Therapeutics. It is based on the delivery of micro-dystrophin (a smaller, but functional, version of dystrophin protein) into muscle cells via adeno-associated virus (AAV) vector. In 2019, Sarepta partnered with Roche to increase the global reach and facilitate accelerated access to SRP-9001 gene therapy to people with DMD living outside of the US.

Throughout Sarepta’s clinical trials for SRP-9001, the NorthStar Ambulatory Assessment (NSAA) has been used as an important and meaningful tool to measure the effect of the gene therapy. The NSAA data collected from the trials provided vital evidence to the assessment committee. This assessment tool was developed by the NorthStar network team and is now being used in over 60 clinical trials across the world. Muscular Dystrophy UK has been funding the work of the NorthStar network for almost two decades and has supported the development of the NSAA.

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