Brasília – Anvisa has issued new details on the ongoing investigation into the gene therapy drug Elevidys® (delandistrogeno moxeparvovexq), while maintaining its temporary suspension. The measure, enacted via Resolution-RE 2,813/2025 on July 25, remains in force following reports of three deaths from acute liver failure in U.S. clinical trials involving the same viral vector technology (AAVrh74).

Elevidys® was approved in Brazil in December 2024 for ambulatory pediatric patients (ages 4 to 7) with Duchenne muscular dystrophy. Only around ten patients have received the treatment so far, with no cases of severe liver toxicity reported locally.

A timeline of the investigation:

August 1: Roche Farma Brasil requested guidance on potential reevaluation.

August 5: Anvisa demanded a detailed technical-scientific report.

August 22: Roche submitted the requested report.

August 29: Anvisa requested additional risk management measures and clarifications.

Anvisa reaffirmed that all decisions are grounded in scientific evidence and regulatory rigor, with patient safety as its top priority.

Source: Anvisa

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