Sarepta Therapeutics and Roche are looking to get approval to continue clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec-rokl) following a review prompted by a patient death.
Three European studies, notably the Envol study (NCT06128564); the Envision study (NCT05881408); and study 104 (NCT06241950), were paused on request by the European Union (EU) reference member country authorities while the independent data monitoring committee (DMC) met to review the adverse event (AE).
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Roche is heading up two of the studies, the Phase II Envol study, which is investigating Elevidys in babies and newborns; and the Phase III Envision, which is evaluating the gene therapy in boys aged eight to 17 years. Sarepta is leading Study 104, a Phase I study evaluating Elevidys in boys aged four to nine years with pre-existing antibodies to a particular serotype.
The independent DMC met on 3 April, a day after the suspensions were announced, and agreed that based on the totality of evidence and the overall benefit-risk profile, dosing in the paused trials could continue without changes to the study protocols.
The companies will submit this data, which it hopes will allow the suspension to be lifted within a week. Evaluation of the submission and the subsequent decision to rescind the temporary halt will follow the EU regulatory process.
On 18 March, Sarepta and Roche announced that a 16-year-old patient in the US who had been treated with Elevidys had died of acute liver failure. The company said it would update the drug’s label to reflect the event. After the death was announced, the company’s stock price took a 27.44% hit, from a $101.35 close on 17 March to a $73.54 close the following day.
Elevidys received approval from the US Food and Drug Administration (FDA) in June 2023 for the treatment of ambulatory and non-ambulatory DMD patients aged four years and older who have a confirmed mutation in the DMD gene. The therapy has not yet been approved in Europe.
The therapy is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy which acts by delivering a transgene that codes for the targeted production of Elevidys micro-dystrophin in skeletal muscle.
Last month, data from studies of several DMD therapies were announced at the 2025 Muscular Dystrophy Association (MDA) meeting in Dallas, Texas, from 16 to 19 March.
Research by GlobalData estimates that across the seven major markets (7MM: US, France, Germany, Italy, Spain, the UK, and Japan), the market for DMD treatments is expected to rise from sales of $2.3bn in 2023 to $5.2bn by 2033, driven heavily by sales of Elevidys and Santhera Pharmaceuticals’ Agamree (vamorolone).
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