Regulatory approval for ELEVIDYS in the UK on hold
Following the European Medicines Agency’s (EMA’s) Committee for Human Medicinal Products (CHMP) decision not to recommend marketing authorisation for ELEVIDYS, Roche has announced that there is currently no regulatory route for approval in the UK.
Approval in the UK
Roche, the company that is delivering ELEVIDYS in the UK, said that since it had been planning to use the EMA’s approval of the treatment to seek market authorisation in the UK, through a mechanism known as the International Recognition Procedure – a mechanism no longer open to them following the EMA’s decision.
In a letter to the community, Roche stated that it will continue its dialogue with the EMA “to explore a potential path forward to make this gene therapy available to individuals living with DMD in the EU.” The outcome of this dialogue would then be used to determine next steps in the UK.
In the UK, the National Institute for Health and Care Excellence (NICE) had begun scoping for a potential appraisal of ELEVIDYS for treating DMD in children aged four to seven years. The scoping had previously been paused at the request of Roche in August 2024 but had been due to resume this summer, with a consultation expected in Autumn 2025. This will now not move ahead.
Roche notes in the community letter that they will continue to communicate with the Medicines and Healthcare products Regulatory Agency, NICE, the Scottish Medicines Consortium, and NHS England and other relevant bodies and “as the path with EMA in the EU becomes clearer,” they will then “assess the next steps” in the UK. However, no timeframes are set out.
Gene therapy
ELEVIDYS (also known as delandistrogene moxeparvovec or dela mox, and SRP-9001) is an adeno-associated virus (AAV) vector-based gene therapy product for ambulatory patients with a confirmed mutation in the DMD gene. It is a one-time treatment designed to treat the underlying cause of DMD.
ELEVIDYS in Europe
The CHMP of the EMA issued a negative opinion regarding ELEVIDYS on 23 July, which means they do not support approval of its conditional marketing authorisation application for the treatment of people with DMD who are ambulatory and aged three to seven years.
European regulators have also requested that Roche and Sarepta (the company manufacturing it in the US) temporarily halt clinical studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). Roche has also paused dosing for the ENVISION study outside Europe.
ELEVIDYS in the US
The Food and Drug Administration (FDA) in the US announced on 28 July that it is recommending the removal of the voluntary hold of ELEVIDYS for ambulatory (walking) patients. This followed an announcement by the FDA on 18 July that it was placing Sarepta Therapeutics investigational gene therapy clinical trials for limb girdle muscular dystrophy on hold following three deaths and safety concerns.
Future of ELEVIDYS
From what we understand, for the time being there will be no further activity towards securing regulatory approval for ELEVIDYS in the UK, until discussions at the European level have concluded, when a way forward could potentially be identified.
The ENVISION study trialling ELEVIDYS in older ambulant and non-ambulant patients also remains on hold. Roche states that that it is committed to ongoing monitoring and evaluation of the benefit-risk profile of ELEVIDYS for people with DMD and will update the patient community as these progress.
Any patient who has already received ELEVIDYS in the UK through clinical trials will continue to be monitored and any concerns you might have in relation to this should be addressed through your clinical team.