The voluntary pauses follow two patient deaths associated with the Duchenne muscular dystrophy gene therapy.
Roche has temporarily halted new deliveries of Elevidys to ambulatory patients in certain countries outside the U.S., marking the latest development in what has been a troubled few months for the Duchenne muscular dystrophy therapy.
The shipment suspension, which a company spokesperson confirmed to BioSpace via email on Wednesday, will apply to new orders of the drug to countries outside the U.S. that have based their own local approvals of Elevidys on regulatory actions by the FDA.
“Based on the totality of available data, Roche believes that the benefit-risk profile is positive in the ambulatory patient population,” the spokesperson told BioSpace, noting that roughly 760 ambulatory patients have been treated with Elevidys “and there have been no treatment-related fatalities” in this group.
“As such, in countries that do not reference the FDA for approval and where Elevidys is approved, Roche will continue to supply and ship Elevidys to enable treatment of ambulatory patients, in consultation with local regulatory authorities,” the spokesperson added.
Discovered and developed by Sarepta Therapeutics, Elevidys is a gene therapy that won accelerated approval in June 2023 and full approval in June 2024. Roche partnered with Sarepta in December 2019, paying $1.15 billion upfront in cash and stock for the right to launch and market Elevidys outside the U.S.
In March this year, Sarepta reported that an Elevidys patient had died after developing acute liver failure, a known complication of the adeno-associated virus vector used to deliver the gene therapy. Another mortality followed in June, prompting the biotech to enact a host of safety measures, including applying a boxed warning to the product. Both deaths occurred in non-ambulatory patients.
Earlier this month, reports of a third patient death surfaced, though this one was linked to a different Sarepta gene therapy, the investigational SRP-9004 being developed for limb-girdle muscular dystrophy.
In response to these deaths, the FDA requested that Sarepta halt all shipments of Elevidys. The company initially refused to comply but on Monday, the biotech announced that it would voluntarily pause U.S. shipments of the gene therapy. As in the case of Sarepta, Roche’s suspension of some ex-U.S. Elevidys shipments is voluntary.
The patient deaths resurface critical questions surrounding Elevidys’ approval. In particular, its full approval in 2024 came despite a failed Phase III confirmatory study in which the gene therapy did not significantly boost functional mobility versus placebo. Despite the failure, Peter Marks, former director of the FDA’s Center for Biologics Evaluation and Research, overruled the agency’s reviewers and pushed for Elevidys’ approval.
Marks’ move has inspired backlash from many quarters, including from Vinay Prasad, his successor at the CBER. “Peter [Marks] was the best ally of Pharma!” Prasad wrote in an X post in March. “He did more for Sarepta than the CEO.”
