Roche Pauses Commercial and Clinical Use of Elevidys After Fatal Liver Failures

By Najat Kantouar

Roche discontinued the commercial and clinical use of Elevidys after two cases of fatal acute liver failure.

The Swiss pharmaceutical company said Sunday that the dosing of non-ambulatory patients, irrespective of their age, is paused in the clinical setting with immediate effect. Enrolment and dosing of these patients will also be halted until additional risk mitigation measures are implemented, it added. Meanwhile, non-ambulatory patients shouldn't receive Elevidys in commercial settings.

The decision follows two cases in non-ambulatory patients of fatal acute liver failure, an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavorable for patients with Duchenne muscular dystrophy who are non-ambulatory, it said.

The company is collaborating with health authorities, investigators and physicians so patient care can be adjusted.

Elevidys has been approved by regulatory authorities in eight Roche region for the treatment of Duchenne muscular dystrophy, including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the United Arab Emirates.

Write to Najat Kantouar at najat.kantouar@wsj.com