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 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.
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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
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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
