Roche Halts Elevidys Dosing in Some Duchenne Patients After Fatalities

Roche (SIX:ROG) shares dropped 2% on Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory patients with Duchenne muscular dystrophy following two deaths caused by acute liver failure.

In a statement released June 15, Roche announced that dosing is suspended in clinical trials and discontinued entirely in commercial use for non-ambulatory patients, regardless of age. The decision follows a reassessment of the benefit-risk profile for this patient group. Acute liver failure, a known risk of adeno-associated virus (AAV)-mediated gene therapies like Elevidys, was confirmed as the cause of death in two patients. Roche reported that approximately 140 non-ambulatory patients worldwide have received Elevidys.

Levi Garraway, Roche’s Chief Medical Officer, said, “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. Patient safety is always our highest priority.”

The European Medicines Agency had already placed temporary holds on three clinical trials—Studies 104, 302 (ENVOL), and 303 (ENVISION)—after the first fatality. Roche confirmed these holds remain in place and has now extended the dosing pause in ENVISION outside Europe. Future commercial use for non-ambulatory patients will require further safety measures.

Elevidys, also known as delandistrogene moxeparvovec, remains approved for ambulatory patients in 10 regions, including the U.S., Japan, and Brazil. Roche co-develops the therapy with Sarepta Therapeutics (NASDAQ:SRPT) under a global agreement. Elevidys is administered as a one-time IV infusion to deliver functional dystrophin protein to muscle cells, aiming to slow disease progression in Duchenne muscular dystrophy, a rare genetic condition affecting 1 in 5,000 boys. Over 900 patients have received the therapy to date.