# Duchenne muscular dystrophy drug regulation in Brazil

> Live situation record from CLSTR: https://clstr.news/situations/duchenne-muscular-dystrophy-drug-regulation-in-brazil
> Updated: 2026-08-24T13:32:00.000Z. Sources: 28. Developments: 2.

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, 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, 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$ 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.

## Claims

- The cost of a single dose of Elevidys is estimated at approximately R$ 17 million. (disputed by 6 sources)
- The cost of a single dose of Elevidys is estimated to be up to R$ 20 million. (disputed by 3 sources)
- Anvisa canceled the conditional registration of the drug Elevidys. (corroborated by 21 sources)
- The cancellation was requested by the pharmaceutical company Roche. (corroborated by 21 sources)
- Available data from the manufacturer did not meet the regulatory requirements to maintain conditional registration. (corroborated by 21 sources)
- Elevidys is indicated for patients with Duchenne Muscular Dystrophy (DMD). (corroborated by 21 sources)
- The cancellation does not change Roche's obligation to monitor patients previously exposed to the drug. (corroborated by 20 sources)
- Two patients in the United States died following episodes of acute liver failure after receiving Elevidys. (corroborated by 3 sources)

## Timeline

### 2026-08-24: Anvisa and Roche cancel Elevidys registration in Brazil

Anvisa and Roche have canceled the conditional registration of the gene therapy Elevidys in Brazil due to insufficient efficacy and safety data. Roche must continue monitoring previously treated patients.

28 sources. https://clstr.news/cluster/anvisa-and-roche-cancel-elevidys-registration-for-duchenne-muscular-dystrophy

### 2026-08-14: Anvisa approves Duvyzat for Duchenne muscular dystrophy

Anvisa has approved Duvyzat (givinostate) in Brazil, an oral treatment designed to slow motor function loss in patients with Duchenne muscular dystrophy.

3 sources. https://clstr.news/cluster/anvisa-approves-duvyzat-for-duchenne-muscular-dystrophy

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Cite as: Duchenne muscular dystrophy drug regulation in Brazil. CLSTR, https://clstr.news/situations/duchenne-muscular-dystrophy-drug-regulation-in-brazil