Roche Restricts ELEVIDYS Use In Non-Ambulatory DMD Patients Following Fatal Liver Failure Cases

Roche has announced immediate new dosing restrictions for ELEVIDYS™ (delandistrogene moxeparvovec) in non-ambulatory patients with Duchenne muscular dystrophy (DMD) due to safety concerns. Effective immediately, non-ambulatory patients will no longer receive ELEVIDYS in commercial settings, and dosing in clinical trials will be paused until updated safety protocols, such as immune modulatory treatments, are implemented.

"We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.”

This decision follows a re-evaluation of the benefit-risk profile after two cases of fatal acute liver failure (ALF) occurred in non-ambulatory DMD patients. ALF is a known risk associated with ELEVIDYS and other AAV-based gene therapies. These incidents prompted a pause in clinical studies across multiple global sites, and regulatory authorities have been notified to adjust ongoing patient care.

The treatment remains available for ambulatory DMD patients of any age, where the benefit-risk assessment continues to be favorable. ELEVIDYS has been approved in eight territories, including Brazil, Japan, and the UAE. Roche and Sarepta Therapeutics jointly oversee clinical development, with Roche leading commercialization outside the United States.