Elevidys

History

2025: Recording deaths of three patients after drug administration

The US Food and Drug Administration has suspended clinical trials of experimental gene therapy for the treatment of cingulate muscular dystrophy and temporarily banned the supply of Elevidis after recording the deaths of three patients during the studies. Sarepta Therapeutics voluntarily stopped all deliveries of the drug to the United States to clarify the circumstances of the incidents. This became known in July 2025.

According to the Pharmacy Times, on July 18, 2025, the FDA requested to suspend the supply of Elevidis for children with Duchenne muscular dystrophy after receiving reports of two deaths among patients. Earlier, the regulator has already suspended clinical trials of gene therapy in connection with the death of one study participant.

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Фото: Freepik

Elevidis is a single intravenous infusion therapy based on adeno-associated virus, designed to eliminate the genetic cause of Duchenne muscular dystrophy. The drug affects mutations in the DMD gene that lead to the absence of dystrophin protein synthesis in muscle tissues.

Treatment is carried out by administering a transgene encoding the synthesis of microdystrophin in the skeletal muscles of patients. The drug is indicated for the treatment of Duchenne muscular dystrophy in ambulatory and nonambulatory patients aged from four years with a confirmed mutation in the corresponding gene.

Drug approval for nonambulatory patients was obtained by the FDA on an expedited basis based on the expression of microdystrophin in skeletal muscle following gene replacement therapy. Further approval depends on confirmation of clinical benefit in additional studies.

Expert Tatyana Gremyakova noted that Duchenne muscular dystrophy is a severe disease that destroys muscle tissue. Any medication showing minimal effect is conditionally approved to reduce research time and provide therapy to children.^[1]

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