[REVISION HISTORY]

Duchenne muscular dystrophy drug regulation in Brazil

Updated 2 times since CLSTR started tracking revisions of this situation.

What changed

2026-08-24 19:46 UTC → 2026-08-24 21:41 UTC · added ~~removed~~

In August 2026, Brazil’s National Health Surveillance Agency (Anvisa) issued two significant regulatory decisions regarding treatments for Duchenne muscular dystrophy (DMD). First, Anvisa approved the registration of Duvyzat (givinostate), an oral medication developed by Multicare Pharmaceuticals. Intended for patients aged 6 and older to be used alongside corticosteroids, clinical studies suggest the drug may slow the loss of motor capacity and preserve muscle function. While approved for commercialization, its availability in pharmacies or the Unified Health System (SUS) remains subject to pricing and further health system evaluations. Conversely, Anvisa and Roche announced the cancellation of the conditional registration for the gene therapy Elevidys (delandistrogene moxeparvovec). The ~~cancellation~~ cancellation, published in the Official Gazette, followed a request from Roche after Anvisa determined that the manufacturer’s data was insufficient to meet regulatory requirements for maintaining conditional authorization. This decision follows a precautionary suspension of the drug’s sale in July ~~2025.~~ 2025, which occurred after reports of acute liver failure in patients treated in the United States. Elevidys is a high-cost treatment, with estimated prices between R$ 10.6 million and R$ 17 20 million per dose. Despite the cancellation, Roche remains legally and medically responsible for the continuous monitoring and follow-up of patients who received the treatment between December 2024 and July 2025, including children in clinical studies and existing access programs. In response to these regulatory shifts, the Brazilian Senate’s Human Rights Commission has scheduled a public hearing to discuss the regulatory situation and public policies for rare diseases.

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