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Sarepta and Roche’s SRP-9001 - FDA Advisory Committee meeting

On 12 May 2023, Sarepta and Roche’s gene therapy for Duchenne muscular dystrophy (DMD) was discussed at an Advisory Committee meeting of the US Food and Drug Administration (FDA). The committee passed a non-binding vote by 8-6 recommending the gene therapy for accelerated approval. This vote will be taken into account by the FDA when they make their final decision at the end of May.

What is SRP-9001?

SRP-9001 (also known as delandistrogene moxeparvovec) is an adeno-associated virus (AAV) vector-based gene therapy product for ambulatory patients with a confirmed mutation in the DMD gene. A company called Sarepta has developed the therapy, and has a licensing agreement with the drug company Roche to launch SRP-9001 outside the United States in places like the United Kingdom. You can read more about the history of SRP-9001 and the relationship between Sarepta and Roche in our update from December 2022 here.

What happened at the meeting?

The Advisory Committee discussed the benefits, risks and uncertainties associated with SRP-9001, with a focus on whether the surrogate endpoints in the application are appropriate. More details can be found in the briefing document published before the committee meeting.

The Advisory Committee held a non-binding vote on whether they support accelerated approval of SRP-9001, which passed with 8 in favour, and 6 against. This vote will be considered by the FDA ahead of its final decision scheduled for the end of May.

Can I watch the meeting?

The meeting took place from 9am – 6pm ET on Friday 12 May 2023, which is 2pm – 11pm in the UK. You can watch it back on YouTube here.

What will happen after the meeting?

The Advisory Committee’s non-binding vote will be considered by the FDA ahead of the scheduled regulatory action date of 29 May 2023. Monday 29 May is a public holiday in the US, so the decision may be made public earlier, on Friday 26 May 2023.

How is this relevant to the UK?

The FDA does not have jurisdiction in the UK. The Medicines and Healthcare products Regulatory Agency (MHRA) will make its own decision once Roche has made an application. However, it is expected that the result of the FDA’s final decision in the US may have ramifications for the availability of SRP-9001 worldwide.

Duchenne UK is in contact with Roche – who have the licence for SRP-9001 in the UK. We will make an update once the FDA have published their final decision.

Published on 12 May 2023

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