Roche to Start Phase III Trial to Broaden Access to Elevidys in Duchenne Muscular Dystrophy

Roche announced plans to initiate a new global, pivotal Phase 3 study of Elevidys (delandistrogene moxeparvovec), with the goal of expanding patient access to the gene therapy for Duchenne muscular dystrophy (DMD). The study is intended to generate additional placebo-controlled data to support a future regulatory resubmission to the European Medicines Agency.

The decision follows feedback from both regulators and the Duchenne patient community, with a focus on addressing unmet needs among ambulatory boys with DMD in Europe and other regions.

What is the objective of the new study?

The planned trial will evaluate the efficacy and safety of Elevidys compared with placebo over a 72-week period in approximately 100 early ambulatory boys with DMD. The primary endpoint will assess change in time to rise from

the floor velocity, a key functional measure and prognostic indicator of disease progression.

Participants initially randomized to the placebo arm will be eligible to receive Elevidys after the primary study period, reflecting an effort to balance rigorous data generation with patient access considerations.

Why is additional data needed?

Elevidys has already received approvals in multiple countries based on data from the EMBARK Phase III study.¹ Results from that trial indicated that treatment with Elevidys resulted in functional improvements and slowed disease progression over 104 weeks (2 years) compared to placebo and found that the therapy is well-tolerated, with manageable safety, showing its potential as a disease-modifying approach.

However, access remains limited in key regions, underscoring the importance of additional clinical evidence to support expanded availability. Roche is seeking to strengthen its clinical evidence package to meet regulatory expectations in additional markets, particularly in Europe.

The new placebo-controlled data are expected to address requirements for a resubmission to the EMA and support broader reimbursement decisions.

Elevidys Indications and Mechanism of Action

Elevidys is a one-time, intravenous gene therapy that uses an adeno-associated virus vector to target the genetic basis of DMD. The therapy works by delivering a functional gene designed to enable muscle cells to produce a shortened form of dystrophin, known as micro-dystrophin, addressing the underlying protein deficiency caused by mutations in the DMD gene.

Elevidys is approved for individuals aged 4 years and older with DMD who have a confirmed DMD gene mutation, including both ambulatory and non-ambulatory patients.In non-ambulatory patients, approval is granted under the FDA’s accelerated approval pathway based on observed micro-dystrophin expression in skeletal muscle.

The most recent regulatory action for Elevidys was in June 2024, when the FDA expanded the approval to include the 4 years and older DMD patient population. The FDA granted a traditional approval to the drug for the treatment of ambulatory patients, with an accelerated approval for non-ambulatory patients.²

For patients with DMD—where treatment options remain limited and disease progression is rapid—the study represents a potential step toward broader global access to gene therapy.

References

1. Francesco Muntoni, F; Eugenio Mercuri, E; Schara Schmidt, U. et al. (2023, April 25). EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP- 9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics (P5-8.012). Neurology.
https://www.neurology.org/doi/10.1212/WNL.0000000000203455

2. Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. Business Wire. June 20, 2024. Accessed April 16, 2026. https://www.businesswire.com/news/home/20240620640445/en/Sarepta-Therapeutics-Announces-Expanded-US-FDA-Approval-of-ELEVIDYS-to-Duchenne-Muscular-Dystrophy-Patients-Ages-4-and-Above