Brasília – The National Health Surveillance Agency ( Anvisa) has updated information on the temporary suspension of Elevidys® (delandistrogene moxeparvovec), a gene therapy authorized in Brazil for ambulatory children aged 4 to 7 with Duchenne muscular dystrophy (DMD). The suspension remains in effect as the agency and the manufacturer continue to review new safety data.

The measure was first adopted after reports of three deaths abroad associated with gene therapies using the same viral vector (AAVrh74), raising concerns over possible liver toxicity. Although no adverse events were reported in Brazil, Anvisa maintained the suspension as a precautionary step.

In October, the agency convened a specialist panel of six experts in DMD, neurology, genetics, and gene therapy to reassess the product’s benefit-risk profile. The group identified safety signals related to hepatic function and immune responses, leading to new requirements for risk management, patient monitoring, and manufacturer accountability.

Anvisa stated that resumption of the therapy’s use will depend on evidence that the product complies with Brazil’s regulatory standards for advanced therapies and that additional safety measures are fully implemented.

Source: Anvisa

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