Roche UK issues a statement following Sarepta’s Press release regarding ELEVIDYS FDA decision

Roche UK issues a statement following Sarepta’s Press release regarding ELEVIDYS FDA decision

Following the press release from Sarepta released on 22nd June 2023 and published on our website here, Roche UK has provided an update to the Duchenne Community.

What has been announced?

The US Food and Drug Administration (FDA) has approved delandistrogene moxeparvovec-rokl (also known as SRP-9001) for the treatment of ambulatory children (those who are able to walk unassisted) aged 4 through 5 years with Duchenne muscular dystrophy (DMD) and a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.

Delandistrogene moxeparvovec-rokl is contraindicated in patients with any deletion in exons 8 and/or 9 in the DMD gene. In the US, delandistrogene moxeparvovec-rokl is the first gene therapy approved for Duchenne.

What does this mean for patients in the UK?

An overview of Roche’s plan for the UK is below:

• 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 actively engaging with the MHRA to determine the best possible route to achieving a potential licence 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 on the NHS.

If you have any other questions about this update, please do not hesitate to contact medinfo.uk@roche.com. Please read the community letter with FAQ’s below:

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.