Roche is suspending deliveries of Elevidys in certain countries, in response to partner Sarepta Therapeutics’ actions in the U.S., due to escalating security worries related to the Duchenne muscular dystrophy gene treatment. The decision means Roche halts access to this therapy in regions where approvals were tied directly to FDA decisions.
The halt pertains to countries where Elevidys approvals were contingent upon the FDA’s endorsements, as stated by a Roche spokeswoman. The list mostly comprises several nations in the Gulf area, including the UAE and Qatar.
Last month, the Swiss pharmaceutical company ceased all distribution of the medicine outside the U.S. in commercial settings for non-ambulatory patients, an arrangement that is still in effect. At that time, Roche emphasized that the drug’s benefit-risk ratio remained favorable for ambulatory patients, but today’s move further halts global access.
As per the Roche spokesperson, Brazil and Japan, whose authorities independently assessed Elevidys without FDA influence, are not impacted by the recent suspension.
The European Medicines Agency began reviewing the Duchenne treatment in the EU in June 2024, but they have not reached a decision yet. For now, Roche halts distribution in all regions where regulators relied on FDA guidance.
In nations impacted by the suspension, no individuals with DMD will have access to the medication, irrespective of their mobility status. On Wednesday, Roche reiterated that it maintains a favorable benefit-risk profile for the ambulatory patient group based on available evidence, even though global health authorities requested that it halts shipments until safety is reassessed.
Roche’s move comes after the FDA asked Sarepta to stop shipments of Elevidys in the country on July 18, following the fatalities of three people who were given the gene treatments produced by the firm.
Sarepta first resisted but later reversed course, as CEO Doug Ingram recognized the importance of maintaining constructive relations with the FDA. This decision set the stage for Roche to follow suit and halt shipments worldwide where FDA influence applies.
Roche confirmed that the latest suspension is temporary but admitted it cannot predict the duration of the delay. In 2019, the company acquired the ex-U.S. license to Elevidys from Sarepta, making its commitment to the drug significant despite these setbacks.
The FDA issued its request to Sarepta after three deaths linked to gene therapy using the AAVrh74 platform were reported, including two teenagers with Duchenne muscular dystrophy treated with Elevidys and a 51-year-old man in a clinical trial for a limb-girdle muscular dystrophy gene therapy. All three patients were immobile and died from severe liver failure.
Roche stated that it continues to believe in the considerable benefit Elevidys offers for ambulatory patients with DMD. The company emphasized its engagement with regulators to understand the FDA’s reasoning for the decision that effectively halts access for patients awaiting treatment.
Due to the limited therapy options for DMD, Elevidys has attracted significant attention. In the first quarter, Roche’s gene therapy generated sales of 43 million Swiss francs, compared to 35 million from Luxturna, a genetic treatment for a rare ocular condition.
Industry analysts note that Roche’s decision to halt shipments highlights the delicate balance between patient safety and the urgent demand for breakthrough therapies. Duchenne muscular dystrophy is a devastating condition with very few treatment options, and every regulatory action has a direct impact on families awaiting new hope.
Healthcare experts suggest that while the suspension may slow halts ’ global rollout, it underscores the need for rigorous long-term safety monitoring of gene therapies. Similar regulatory pauses have been seen before in pioneering fields, often followed by additional data reviews and revised protocols that pave the way for therapies to safely return to market.
Looking forward, Roche is expected to provide updated clinical findings and collaborate with independent experts to address the FDA’s concerns. If positive data emerges, the current halts could be lifted, potentially restoring patient access and reinforcing confidence in the therapy’s long-term promise.
