By Brazil Stock Guide – Brazil’s health regulator Anvisa said it is still reviewing additional information submitted by Roche Farma Brasil on Elevidys, a gene therapy used to treat Duchenne muscular dystrophy. Sales and use of the treatment remain temporarily suspended in Brazil.
The agency also said it is waiting for further data requested from the company before moving ahead with its review. Elevidys is estimated to cost about R$20 million per patient, making it one of the most expensive therapies ever assessed in the Brazilian market.
The review includes changes to clinical protocols, adjustments to the risk-management plan and refinements to long-term monitoring strategies. According to Anvisa, the measures are intended to ensure the safe and responsible use of the therapy in Brazil.
Additional Data
In February, Roche submitted a new proposal for a commitment agreement with the regulator. After a technical review, Anvisa requested specific data required under the current agreement.
The information includes long-term follow-up data from clinical-trial participants, as well as interim efficacy data for the product.
Anvisa also requested updated and consolidated safety information, with a focus on acute liver failure, deaths and other serious adverse events. The agency also asked for causality analyses, proposed improvements to the risk-management plan and additional risk-mitigation and monitoring measures, especially for liver function.
Conditional Approval
Elevidys was approved in Brazil in December 2024 on an exceptional basis, with the requirement that Roche submit additional efficacy and safety data.
According to Anvisa, the approval took into account the specific characteristics of advanced-therapy products, the rarity of the disease and uncertainties surrounding the available clinical evidence. The conditional approval was granted under RDC 505/2021, Brazil’s regulatory framework for such products.
In Brazil, the therapy was authorized for the treatment of Duchenne muscular dystrophy in ambulatory patients — children who are still able to walk, with or without assistance — between the ages of 4 and 7, subject to specific clinical and laboratory eligibility criteria.
Suspension Still in Place
In July 2025, Anvisa temporarily suspended the sale and use of Elevidys in Brazil. The decision followed new international information on serious cases of acute liver failure, including fatal cases after administration of the treatment.
The agency also cited similar findings involving other gene therapies that use comparable technologies and mechanisms of action through analogous vector platforms.
The temporary suspension remains in place while Anvisa completes its additional technical reviews and continues discussions with Roche, the company responsible for the product’s registration in Brazil.
Global Reviews Differ
Anvisa said public documents show differences among foreign regulatory authorities recognized by the Brazilian agency in their assessments of Elevidys’ benefit-risk balance.
The therapy is authorized in the United States and Japan. The European Medicines Agency, however, refused marketing authorization for the product based on its assessment of the primary endpoint in the clinical study.
In October 2025, Anvisa held technical discussions with specialists through an independent scientific panel. According to the agency, the discussions identified additional issues related to liver-safety profile and the immune response to the viral vector used in the therapy.
No Deadline Yet
Anvisa said the Elevidys review is being handled as a priority, given the severity of Duchenne muscular dystrophy, its clinical and social impact, and the need to protect children and adolescents.
The agency also said it cannot provide a timeline for completing the review. The final decision will depend on the quality of the information submitted by Roche and on Anvisa’s technical assessment.
At the end of the process, the regulator will decide whether to maintain, change or lift the precautionary suspension of Elevidys in Brazil.
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