Defeat Duchenne Canada expresses our sympathy for the family impacted by the untimely death of an unnamed teen with Duchenne muscular dystrophy (DMD) who died shortly after receiving Sarepta’s ELEVIDYS® (delandistrogene moxeparvovec-rokl) due to acute liver failure. Elevidys is approved in the United States as treatment for all people with DMD who are at least four years of age with a confirmed mutation in the dystrophin gene, except for those who have any deletion in exon 8 and/or exon 9 in the DMD gene. It is not currently approved in Canada, although Canadians may have participated in the clinical trials.
“On behalf of Defeat Duchenne Canada and all Canadian families affected by Duchenne, I wish to extend sincerest condolences to the family that has recently suffered the heartbreaking loss of their teenage child. While the development of new, effective treatments is critical, we understand there can be risks and that sometimes, other unrelated illnesses can increase those risks. In this case, while more information may be available at a later date, it does not lessen the pain of the family that has suffered this immense loss. Our thoughts remain with them at this very difficult time.”
Lisa McCoy, Chief Executive Officer, Defeat Duchenne Canada
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 the side effects, even if they do not think that the medicine caused the side effect (for example, 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 person with DMD who passed away after treatment with Elevidys, the doctor noted that the deceased patient also had a recent cytomegalovirus (CMV) infection. A CMV is a common herpes-related virus that can cause fever, chills, sore throat, fatigue, and can impact the liver. This infection could have impacted the person’s liver.
“We are collecting additional information, but based on the current information from the treating physician, including the time since treatment and the clinical course, we cannot rule out the possibility that Elevidys was a contributing factor.”
Spokesperson for Sarepta, manufacturer and marketer of Elevidys.
People who receive Elevidys are always monitored for liver injuries for the first three months after treatment. Acute liver injury is among the warnings and precautions included in the prescribing information for Elevidys.
Please see the full news article below for additional details.
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