Increasing efficacy of Elevidys in DMD

L’essai de phase IIIAu cours d’un essai clinique de phase III, un médicament, pour lequel on a déterminé lors d’essais antérieurs l’innocuité et le dosage optimum (essais de phase I et II), est administré à un grand groupe de malades, sur une longue durée, dans le but d’évaluer son efficacité thérapeutique en la comparant à celle d’un traitement de référence ou un placebo. Il permet aussi de mettre en évidence les interactions indésirables et les effets secondaires du traitement à moyen terme. Au terme de cet essai, le médicament peut obtenir une autorisation de mise sur le marché. EMBARK avait présenté des résultats non significatifs contre placebo pour le critère principal à un an, ce qui avait valu un avis défavorable de l’Agence européenne du médicament (EMA) à la commercialisation de l’Elevidys dans la dystrophie musculaire de Duchenne (DMD).

The phase III EMBARK trial showed non-significant results compared to placebo for the primary endpoint at one year, which led to an unfavorable opinion from the European Medicines Agency (EMA) on the marketing of Elevidys for Duchenne muscular dystrophy (DMD).

New results, this time significant, show longer-term stabilization or slowing of disease progression in treated boys aged 4 to 8 years who are able to walk, compared to a matched external control group.

• Treated patients achieved higher NSAA scores than those measured at the start of the study: a 2.63-point improvement at two years (compared to -0.25 points for the control group). Treated children got up from the floor faster (0.65 vs. 2.71 seconds) and were faster at walking 10 meters (-0.04 vs. 1.32 seconds).
• Three years after treatment, children showed at least a 70% reduction in the rate of decline compared to the control group.
• No new safety signals were observed.

‘Patients treated with Elevidys showed an increasing therapeutic effect over time, with the functional gap compared to the control group widening significantly between the second and third years’ said Sarepta Therapeutics.

Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. Mendell JR, Muntoni F, McDonald CM et al. Neurol Ther. 2026 Jan 10.

 

Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory Duchenne Patients. Sarepta Therapeutics. 26 January 2026 press release.