Roche is pausing shipments of Elevidys in some countries, following partner Sarepta Therapeutics’ move in the U.S., as safety concerns mount over the Duchenne muscular dystrophy (DMD) gene therapy.
The suspension covers countries where approvals for Elevidys were based on the FDA’s green lights, a Roche spokesperson told Fierce Pharma. The list mainly includes several states in the Gulf region, such as the United Arab Emirates and Qatar.
The Swiss drugmaker last month stopped all ex-U.S. distribution of the drug in the commercial setting for non-ambulatory patients, a policy that remains in place. At the time of that decision, Roche said the drug’s “benefit-risk ratio remains positive in the ambulatory patient population.”
Brazil and Japan, where regulators made their own decisions on Elevidys independent of the FDA, are not affected by the latest pause, according to the Roche spokesperson.
In the EU, the European Medicines Agency initiated a review of the DMD therapy in June 2024 but has not issued a verdict.
In countries affected by the latest pause, no DMD patients will be able to access the therapy—regardless of their ability to walk. Wednesday, Roche said it still believes “the benefit-risk profile is positive in the ambulatory patient population” based on the totality of available data.
Roche’s decision follows a request from the FDA to Sarepta on July 18 to halt Elevidys shipments in the U.S. after the deaths of three patients who had received the company’s gene therapies.
Sarepta initially stood its ground but soon reversed course, as CEO Doug Ingram acknowledged the importance for the Massachusetts biotech to maintain “a productive and positive working relationship with FDA.”
Roche said the latest halt is temporary but that it cannot speculate on how long the pause will be in effect. The Swiss pharma licensed ex-U.S. rights to Elevidys from Sarepta in 2019.
The FDA sent the request to Sarepta after reports of three deaths among patients who had received a gene therapy based on the biotech’s AAVrh74 platform, including two DMD teenagers who’d received Elevidys and one 51-year-old man who received an investigational limb-girdle muscular dystrophy gene therapy in a clinical trial. All three patients were non-ambulatory and died from acute liver failure.
After the second death of an Elevidys patient, reported in June, Sarepta suspended Elevidys supplies to non-ambulatory patients in the U.S. At the time, Roche said it had “discontinued” the drug in non-ambulatory populations outside the U.S.
“Roche remains confident in the significant value Elevidys can bring to ambulatory patients with DMD,” the company said Wednesday in a statement to Fierce Pharma.
“Roche is working to understand why the FDA has requested that shipments be paused in the ambulatory patient population, and is in contact with the appropriate health authorities globally to determine next potential steps,” the company added.
As treatment options for DMD are limited, Elevidys has garnered much interest. For Roche, the gene therapy brought in 43 million Swiss francs in sales in the first quarter, versus 35 million Swiss francs from Luxturna, a gene therapy for a rare eye disease.