Brazil's National Health Surveillance Agency (ANVISA) announced the temporary suspension of the marketing, distribution, importation, manufacturing, advertising, and use of the gene therapy drug ELEVIDYS® (the delandistrogen moxeparvovek).

The preventive measure was adopted following the reporting of three deaths from acute liver failure in the United States in patients treated with gene therapies based on the AAVrh74 vector.

The health authority reinforces its commitment to protecting public health and ensuring safe access to advanced therapies, emphasizing that any indication of risk in an innovative and complex product requires immediate regulatory action.

WHOM DOES IT IMPACT?

The direct impact is on pediatric patients with DMD, healthcare professionals involved in their treatment, importers and manufacturers, research centers, and hospitals that administer gene therapies.

CONCLUSION:

The temporary suspension of ELEVIDYS® in Brazil reflects a necessary precautionary measure considering international signals of serious risk, prioritizing the safety of pediatric patients with Duchenne muscular dystrophy. Although no fatal cases have been reported in the country and its use is highly restricted, ANVISA's action seeks to reevaluate the benefit-risk ratio before resuming access, maintaining confidence in the regulation and ensuring the protection of this vulnerable population.