Key points:
- FDA approves ELEVIDYS for DMD patients aged 4 and above, including both ambulatory and non-ambulatory individuals.
- Traditional approval granted for ambulatory patients, while accelerated approval given for non-ambulatory patients.
- Sarepta commits to conducting a confirmatory trial for non-ambulatory patients to verify clinical benefits.
The expanded label for ELEVIDYS (delandistrogene moxeparvovec-rokl) now includes individuals with DMD who are at least 4 years old, regardless of their ability to walk. This decision marks a crucial advancement in gene therapy for this devastating rare disease.
Doug Ingram, Sarepta’s CEO, hailed the approval as “a defining moment for the Duchenne community” and “a watershed occasion for the promise of gene therapy.” Dr. Jerry Mendell, co-inventor of ELEVIDYS, emphasized the significance of this expansion, stating it provides clinicians with a treatment option for the majority of boys and young men living with Duchenne.
To fulfill the requirements of the accelerated approval pathway, Sarepta has initiated the ENVISION study, a global Phase 3 trial, to confirm the clinical benefits of ELEVIDYS in non-ambulatory and older ambulatory DMD patients.
This expanded approval not only represents a major scientific achievement but also offers new hope to a broader range of DMD patients and their families.
