Roche Updates Elevidys™ Gene Therapy Safety for Non-Ambulatory Duchenne Patients

In a recent announcement from BASEL, Switzerland dated June 15, 2025, Roche has implemented new dosing restrictions for ELEVIDYS™ (delandistrogene moxeparvovec), specifically targeting non-ambulatory patients suffering from Duchenne muscular dystrophy (DMD). This decision impacts both clinical trials and commercial applications, with non-ambulatory patients no longer receiving the treatment in the commercial market. Meanwhile, ongoing clinical trials will pause the enrolment and dosing of non-ambulatory patients until new risk mitigation strategies, such as immune modulatory treatments, are integrated into the study protocols. Roche is actively communicating these changes to health authorities, investigators, and medical practitioners to ensure immediate adaptation in patient care.

This decisive action follows a detailed evaluation of two instances of fatal acute liver failure (ALF) in non-ambulatory patients treated with Elevidys. These incidents have led to the re-evaluation of the benefit-risk profile of the drug in non-ambulatory DMD patients, now considered unfavorable. Consequently, the treatment will continue only for ambulatory DMD patients, where the benefit-risk assessment remains positive.

Roche’s Chief Medical Officer and Head of Global Product Development, Levi Garraway, M.D., Ph.D., expressed deep sorrow over the loss of two patients and emphasized the urgency in mitigating associated risks with Elevidys usage. Garraway reiterated the company's commitment to patient safety and announced the immediate cessation of Elevidys treatment for non-ambulatory patients.

Duchenne muscular dystrophy is a rare genetic disorder characterized by rapid muscle degeneration beginning in early childhood. It predominantly affects males, with about one in 5,000 boys worldwide diagnosed with Duchenne. The disease eventually leads to loss of mobility and upper limb, lung, and cardiac functions.

Out of approximately 140 non-ambulatory patients who received Elevidys globally, two experienced fatal ALF. Following the first ALF case, European regulators instructed Roche and its partner Sarepta Therapeutics to impose temporary holds on several Elevidys studies, including studies 104 (NCT06241950), 302 (ENVOL, NCT06128564), and 303 (ENVISION Study 303, NCT05310071). These holds remain in place, and outside Europe, dosing is on hold for the ENVISION trial. The new dosing restrictions will apply to any future commercial dosing of non-ambulatory patients.

Elevidys has gained approval in eight Roche territories for DMD treatment, including countries like Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche and Sarepta Therapeutics entered a global collaboration to commercialize Elevidys outside the U.S. while jointly managing its clinical studies. Roche leads the ENVOL study, whereas Sarepta manages the other studies.

Elevidys represents a groundbreaking treatment for Duchenne, being the first and only gene therapy approved for the condition. Administered as a one-time intravenous dose, it aims to address Duchenne's root cause by instructing cells to produce Elevidys-dystrophin in crucial muscles, thereby slowing disease progression. The therapy utilizes adeno-associated virus vector technology to deliver its components efficiently to all muscle types relevant to Duchenne treatment.

Elevidys is undergoing extensive clinical trials to assess its efficacy across diverse demographics with Duchenne, encompassing various ages, ambulatory statuses, and genetic mutations. To date, over 900 individuals have received Elevidys under Roche’s clinical development program and in real-world applications. The therapy has received approval for DMD treatment in 10 countries worldwide, including the U.S. and Japan, with development spearheaded by Roche in collaboration with Sarepta.