The European Medicines Agency (EMA), who oversees the drug approval process for the European Union, has put three clinical trials of Sarepta/Roche’s ELEVIDYS® (delandistrogene moxeparvovec-rokl) for people with Duchenne muscular dystrophy (DMD) on hold while the previously reported death of an American teenager who had just taken the drug is investigated. The trials on hold are SRP-9001-104 (NCT06241950), BN43881 (NCT06128564), and ENVISION (NCT05881408).
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.
Sarepta/Roche issued a letter to the community providing additional information about this clinical hold. Please see the full letter below for additional details.
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