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Investing.com -- Roche (SIX:ROG) shares fell 2% Monday after the company paused dosing of its gene therapy Elevidys in non-ambulatory patients with Duchenne muscular dystrophy, following two reported cases of fatal acute liver failure.
In a June 15 statement, Roche said the decision affects both commercial and clinical use of the treatment in this patient population, regardless of age.
Dosing is suspended in clinical trials, and discontinued entirely in commercial settings.
The move comes after a reassessment of Elevidys’ benefit-risk profile in non-ambulatory patients.
Acute liver failure, a known risk of adeno-associated virus (AAV)-mediated gene therapies such as Elevidys, was cited as the cause of death in two patients.
To date, approximately 140 non-ambulatory patients have been treated with Elevidys globally, Roche 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,” said Levi Garraway, Roche’s chief medical officer. “Patient safety is always our highest priority.”
The European Medicines Agency had already placed temporary clinical holds on three ongoing Elevidys trials, Studies 104, 302 (ENVOL), and 303 (ENVISION), after the first fatality.
Roche said these holds remain in place, and dosing for ENVISION is now also paused outside Europe. Future commercial use in non-ambulatory patients will not proceed unless additional risk mitigation is implemented.
Elevidys, also known as delandistrogene moxeparvovec, remains available for ambulatory patients, where the benefit-risk profile continues to be viewed as favorable. Roche stated that treatment guidance for this group remains unchanged.
The therapy is approved in 10 countries and territories, including Japan, Brazil, and the U.S. Roche co-develops Elevidys with Sarepta Therapeutics (NASDAQ:SRPT) under a global collaboration agreement signed in 2019. While Roche sponsors the ENVOL study, Sarepta leads other clinical trials for the drug.
Elevidys is administered as a one-time intravenous dose, intended to deliver functional dystrophin protein to muscle cells to slow the progression of Duchenne muscular dystrophy, a rare genetic disorder that causes progressive muscle loss.
The disease affects 1 in 5,000 boys globally and typically leads to loss of mobility and premature death.
Across its clinical program and real-world use, more than 900 individuals have received Elevidys.
Roche said it is working with regulators, investigators, and healthcare providers to inform stakeholders and adjust patient care as needed.