Roche UK issues statement to the Duchenne Community following Sarepta’s Update on Regulatory Review of SRP-9001 gene therapy.

Roche UK issues statement to the Duchenne Community following Sarepta’s Update on Regulatory Review of SRP-9001 gene therapy.

Following the update from Sarepta released on 24th May 2023 and published on our website here, Roche have provided an update to the Duchenne Community.

What has been announced?

Following the U.S. Food and Drug Administration (FDA) advisory committee meeting, Sarepta had
an informal telephone conference with the FDA to discuss the next regulatory steps for the
accelerated Biologics License Application (BLA) review. The FDA has indicated that, subject to the
completion of the review of delandistrogene moxeparvovec for the treatment of Duchenne
muscular dystrophy, it is working toward potentially granting an accelerated approval, initially for
use in Duchenne patients aged 4-5 years old.

What does this mean for patients in the UK?

Different health authorities act independently, following distinct processes with different filing
requirements. Although their ultimate goals are the same for their geographical regions, they
have distinct regulations and procedures, meaning there are differences in how medicines are
approved.

The MHRA is the health authority in the UK and is responsible for issuing Marketing Authorisations
for medicines. We are engaging with the MHRA to determine the best possible route to achieving
a potential future license for delandistrogene moxeparvovec. It is worth noting that in the UK,
regulatory approval is only the first step. Further assessments of clinical and cost-effectiveness
by the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines
Consortium (SMC) must also be undertaken before a medicine is available to patients. We will
share updates in due course.

Read the full statement here:

Do you want to know more?

• To find out more about Duchenne science, gain crucial knowledge and support, please join us on our Science on Tour workshops. We are coming to 30 locations across the UK in 2023 – book your FREE place here now. 
• SAVE THE DATE: Action Duchenne’s International Conference 2023 will be held on the 10th and 11th November and is an amazing opportunity to meet with those involved in every aspect of Duchenne, from families, clinicians, researchers and pharmaceutical companies.