Unfavorable opinion on marketing authorisation and suspension of clinical trials in Europe for Elevidys in DMD

Over the summer, the European Medicines Agency (EMA) issued an unfavourable opinion on the conditional marketing authorisation of Elevidys, a microdystrophin gene therapy for ambulatory patients aged 3 to 7 years with Duchenne muscular dystrophy (DMD).

• This opinion, which must be ratified by the European Commission, is based on insufficient data on the treatment’s efficacy on motor function at one year in the EMBARK trial (non-significant difference of +0.65 NSAA points between the treatment and placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo. groups).
• The efficacy results for Elevidys need to be consolidated, according to two publications that analysed several trials of the drug. They conclude that additional efficacy data (particularly in real life) needs to be collected and that the treatment’s side effects related to the immune system need to be closely monitored.
• Following the deaths of three patients treated with Elevidys, including two non-ambulatory patients (due to a liver problem related to the product) and one ambulatory patient (which was not related to the treatment), Roche, the laboratory developing the product outside the United States, suspended its clinical trials in Europe for all patients.
• Sarepta Therapeutics has resumed supplying Elevidys to ambulatory patients, but has discontinued supply to non-ambulatory patients.

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus: Report of the AAN Guidelines Subcommittee. Oskoui M, Caller TA, Parsons JA et al. Neurology. 2025 Jun 10;104(11)

 

Efficacy of delandistrogene moxeparvovec on Duchenne muscular dystrophy: a systematic review and meta-analysis. Pascual-Morena C, Patiño-Cardona S, Martínez-García I et al. Hum Genet. 2025 Jun 26.

 

Elevidys overview – EMA, 24 July 2025

 

Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU – Roche. 25 July 2025 press release

 

FDA Informs Sarepta That It Recommends That Sarepta Remove Its Pause and Resume Shipments of ELEVIDYS for Ambulatory Individuals With Duchenne Muscular Dystrophy – Sarepta Therapeutics. 28 July 2025 press release