Roche has paused both commercial and clinical use of its gene therapy Elevidys for non-ambulatory patients with Duchenne muscular dystrophy (DMD) following two reported cases of fatal acute liver failure, the Wall Street Journal reports.
In a statement released Sunday, the Swiss pharmaceutical company announced the immediate suspension of Elevidys dosing and enrollment for non-ambulatory patients in clinical trials, regardless of age. The company also advised against administering the therapy in commercial settings for this patient group until further safety measures are put in place.
The decision was made after a reassessment of the therapy’s benefit-risk profile, which was determined to be unfavorable for non-ambulatory DMD patients in light of the recent fatalities. Acute liver failure is a known risk associated with Elevidys and other AAV-based gene therapies, Roche noted.
The company said it is working closely with health authorities, investigators, and healthcare providers to evaluate next steps and ensure patient care is managed appropriately.
Elevidys has received regulatory approval for the treatment of Duchenne muscular dystrophy in several countries, including Japan, Brazil, Israel, Bahrain, Kuwait, Oman, Qatar, and the United Arab Emirates.
Roche has not indicated changes to Elevidys availability for ambulatory patients at this time.
