Access to treatments

Path to gene therapy approval in the UK – Sarepta and Roche’s SRP-9001

Following the exciting news that the Food and Drug Administration (FDA) in the United States has accepted for filing and granted for priority review Sarepta’s gene therapy product, SRP-9001, many DMD parents have been in touch us asking what plans there are for possible approval in the UK.  

We have been talking to Sarepta and are delighted to report discussions are already underway between the drug company’s partner and the UK regulatory authority about how best to proceed. This promising news is just the beginning of a process. 

What’s happening in the USA?  

The priority review granted by the FDA allows for shorter timeframes for approval than the standard ten months. The review has a regulatory action date of 29 May 2023. The application is for treatment of ambulant individuals with Duchenne muscular dystrophy (DMD). 

What’s happening in the UK? 

Sarepta has entered a licensing agreement with the drug company Roche to launch SRP-9001 outside of the United States. Roche have issued the following statement regarding UK approval: 

“Roche recognises that there is a significant unmet medical need for those living with DMD. We are actively working to determine the most appropriate path for submission to bring SRP-9001 to patients in the UK, and have already engaged in an early dialogue with the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK. We look forward to providing further updates about the SRP-9001 access pathway as they become available.” 

The MHRA and FDA are different authorities and act independently. They have distinct regulations and processes. In the UK, MHRA approval is also only the first step. Further assessments of clinical and cost effectiveness are made by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) in Scotland before a medicine is made available to patients. 

What is gene therapy? 

DMD is a genetic disease. Gene therapy aims to correct the genetic fault that leads to the lack of dystrophin in muscles which causes them to gradually weaken. 

Read more about gene therapy here. 

SRP-9001 – Embark trial. 

A phase 3 trial for SRP-9001 is underway. The first boy in the UK, Charlie Anderson, on the trial was recruited through DMD Hub Central Recruitment Project 

Emily Reuben is the CEO of Duchenne UK: 

“When we began our fight against Duchenne, we were told gene therapy would not be available in our sons’ lifetimes. Now not only are several trials underway, but the approval process for one treatment has begun. This is just the start of the process, and there is no guarantee SRP-9001 will be approved in any jurisdiction. But we are confident gene therapy will provide a treatment for Duchenne that address the root cause of the disease, rather than just its’ symptoms.” 

Please do not hesitate to contact Duchenne UK should you have further questions. Join our Parent Information Day on 4 March 2023 in London for more guidance on the latest research and treatments.  

Published on 9 December 2022

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