Sarepta Facing Pressure - Roche Refuses to Collaborate
In a significant development for the treatment of Duchenne muscular dystrophy (DMD), the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a negative opinion on Elevidys™ (delandistrogene moxeparvovec) gene therapy by Sarepta Therapeutics. This decision, announced in July 2025, follows a reassessment of the benefit-risk profile, particularly due to two reported cases of fatal acute liver failure in non-ambulatory patients receiving the therapy.
As a result, dosing of non-ambulatory patients with Elevidys has been paused in clinical settings and discontinued in commercial use across Europe. However, the benefit-risk profile for ambulatory DMD patients remains positive, and treatment guidance for this group has not changed, meaning the therapy is still considered viable for ambulatory patients under current guidelines.
From a regulatory and market access standpoint, Elevidys remains a restricted and closely monitored therapy. It appears listed with prior authorization requirements in certain health coverage policies as of mid-2025. This reflects an ongoing cautious approach pending further safety evaluations.
The future implications of this decision include intensified monitoring of liver safety risks with Elevidys and potential modification of its approved indications—likely continuing only for ambulatory DMD patients unless further data support broader use. Sarepta Therapeutics and regulatory agencies will need to collaborate closely on risk mitigation and patient management strategies.
Meanwhile, Evrysdi, a similar gene therapy developed by Sarepta and marketed in the US, has been the subject of a separate regulatory decision. The CHMP has expressed a negative opinion on the marketing authorization application for Roche's Evrysdi (delandistrogene moxeparvovec) for outpatient patients aged 3 to 7 years with DMD.
Roche, which holds the rights to Evrysdi outside the US, had applied for the authorization of Evrysdi for outpatient patients with DMD. More than 900 people with DMD, including 760 outpatients, have been treated with Evrysdi, according to Roche. The authorization process for Evrysdi has been overshadowed by recent deaths linked to the therapy, which could potentially hinder the availability of Evrysdi for outpatient patients with DMD in Europe.
Roche temporarily stopped the delivery of the gene therapy in some countries outside the US. The future of Evrysdi in Europe remains uncertain, with regulatory decisions and safety evaluations ongoing.
In summary, the CHMP’s negative opinion on Elevidys and the potential negative opinion on Evrysdi restrict their use in Europe, particularly halting treatment in non-ambulatory patients due to safety concerns. Treatment for ambulatory patients continues with no change in guidelines, allowing ongoing access and evaluation. Health authorities and the respective companies are taking steps to manage patient safety and regulatory compliance. The future outlook depends on additional safety and efficacy data, with potential impact on labeling, usage restrictions, and market acceptance in Europe.
Science and health-and-wellness intersect in the field of medical-conditions, such as Duchenne muscular dystrophy (DMD), where new treatments like Elevidys™ and Evrysdi are being developed. However, the recent decisions by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) have raised concerns about the safety of these gene therapies, leading to restricted use and close monitoring. Finance and investing are also involved, as the regulatory decisions can impact market access and the future development of these potential treatments.
