Update on Sarepta and Roche's SRP-9001 gene therapy
Today, Wednesday 24 May 2023, Sarepta announced the FDA’s decision date to grant accelerated approval to their gene therapy SRP-9001 has been pushed back to 22 June 2023, but that the FDA has indicated they are working towards granting accelerated approval for SRP-9001 in 4 and 5-year-olds.
You can read the investor statement from Sarepta here.
You can also read Sarepta’s US community bulletin here.
Roche’s community update is here.
This announcement by Sarepta Therapeutics comes with several caveats, and the United States Food and Drug Administration (FDA) are yet to directly comment. Approval is still subject to the completion of the FDA’s review of the Biologics License Application (BLA) for SRP-9001 (delandistrogene moxeparvovec).
In addition, this news and the potential approval only applies in the United States. Britain’s drug approval body, the Medicines and Healthcare products Regulatory Agency (MHRA), will make a decision separately based upon an application by Roche, the company with the licence for SRP-9001 in the UK.
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.
Didn’t the FDA already approve SRP-9001 this month?
On 12 May 2023, the FDA’s advisory committee voted by a narrow margin of 8-6 to recommend the treatment for accelerated approval. That vote was non-binding and non-final, but will be taken into account by the FDA in their decision-making. You can read our article about the advisory committee vote in our mid-May update here.
What is a BLA, the ‘regulatory action date’, and why has it been pushed back?
A Biologics License Application (BLA) is a submission by a company to the FDA that contains specific information on a product they want to market in the US. If the information provided meets FDA requirements, the application is approved and a license is issued to the company.
‘Regulatory action date’ is the term used by the FDA to refer to when it should conclude their review of drug approval submissions like BLAs. The original regulatory action date for the BLA for SRP-9001 was 29 May 2023. This has now been pushed back to 22 June 2023.
In their announcement, Sarepta said the FDA requires modest additional time to complete the BLA review, including final label negotiations and postmarketing commitment discussions, and this is why the date has been pushed back to June.
When will patients over 4 and 5-years-old get access in the US, and what is EMBARK?
Sarepta’s statement says that the FDA’s indicated approval will initially only be for 4 and 5-year-olds. It goes on to say that EMBARK is the proposed confirmatory study, and that non-age-restricted access for SRP-9001 will be dependent on the EMBARK trial meeting its objectives.
The EMBARK trial (Study 301, NCT05096221) is a global, phase 3, randomised, double-blind, placebo-controlled study. 120 patients are taking part in EMBARK, and the primary outcome measure is based on changes to North Star scores. It is also the first trial of SRP-9001 to have UK clinical trials sites. The first boy in the UK on the trial, Charlie Anderson, was recruited through DMD Hub Central Recruitment Project.
The results of EMBARK are currently expected in the fourth quarter of 2023, but that could change. You can read more about EMBARK on the DMD Hub here.
What does this mean for the UK?
In the short term, this decision by the FDA does not affect the availability of SRP-9001 in the UK. British regulatory authorities will make their own decision in due course based on Roche’s application.
Roche’s update, also linked to at the top of the article, can be found here.
When will SRP-9001 come to the UK?
Currently, that is very hard to predict, and will hinge on Roche’s application with the MHRA, and then after that, with decisions by the National Institute for Health and Care Excellence (NICE).
There have been no announcements on where Roche is in that process, or what route to approval SRP-9001 will take, but Duchenne UK understands that Roche and the MHRA are currently in discussions about SRP-9001.
The default notes for editors here.