The Medicines & Healthcare products Regulatory Agency (MHRA), who oversee the drug approval process in the United Kingdom (U.K.), has lifted the temporary clinical hold they previously put on the Phase 3 ENVISION, study SRP-9001-303 (NCT05881408), of Sarepta/Roche’s ELEVIDYS^® (delandistrogene moxeparvovec-rokl) for people with Duchenne muscular dystrophy (DMD) while the death of an American teen who had received treatment was investigated.

Elevidys is given to people with DMD with a single intravenous (IV) infusion. It is a gene therapy that uses an adeno-associated viral (AAV) vector to deliver a smaller version of the dystrophin gene called a “microdystrophin.” The AAV is used to deliver the healthy gene, with the intention of allowing a person with DMD to create dystrophin with this new healthy gene. Unfortunately, there are side effects called “adverse events” that are known to occur with AAV gene therapies. When someone receives a treatment and there is a side effect, the doctor who administered the medicine has to report this side effect, even if they do not think that the medicine was the cause. An example of this may be if someone receiving a medicine developed a headache. The doctor would report the headache as a side effect, even if the person has the flu which could be the cause of the headache.

In the case of the American teen, it is not confirmed that the death was due to treatment with ELEVIDYS. However, because the death occurred too soon after infusion, there will be an investigation into whether the death is related to treatment.

Please see the full Press Release below for additional details.

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