Brazil’s National Health Surveillance Agency (ANVISA) has cancelled the registration of Elevidys, a gene therapy used to treat Duchenne muscular dystrophy (DMD), following an assessment that the available efficacy and safety data were insufficient for definitive authorization.
The decision was announced on August 24, 2026. With the cancellation published in Brazil’s Official Gazette (DOU), the importation, distribution, and commercialization of Elevidys are officially suspended in the country.
Elevidys had been granted conditional authorization in Brazil since December 2024. The therapy was indicated for children aged 4 to 7 with Duchenne muscular dystrophy who retained the ability to walk independently and did not have pre-existing antibodies against the viral vector used for administration.
According to the report, ANVISA’s decision was based on its assessment that the data submitted regarding the therapy’s efficacy and safety were insufficient to support definitive authorization.
Elevidys was not incorporated into Brazil’s Unified Health System (SUS). Instead, the treatment had been obtained in the country through legal proceedings and was used as a gene therapy for children with Duchenne muscular dystrophy.
Following the cancellation, ANVISA will still determine how cases involving patients who had already begun the process of purchasing or receiving the therapy will be handled.
The cancellation therefore affects the regulatory status of Elevidys in Brazil, suspending its importation, distribution, and commercialization following the agency’s assessment of the available efficacy and safety data.
Elevidys is a one-time gene therapy used for Duchenne muscular dystrophy. In Brazil, its previous authorization covered a specific group of children with the condition under the criteria established for its conditional authorization.
Learn More: Frequently Asked Questions About Elevidys
