Brazil's health regulatory agency Anvisa on Monday (24) canceled the conditional registration of Elevidys (delandistrogene moxeparvovec), a gene therapy used to treat Duchenne muscular dystrophy and ranked among the most expensive medicines in the world, with a single dose estimated at up to R$ 20 million. The decision was published in the Federal Official Gazette and, according to the agency, followed a request from the manufacturer, Roche.

In a joint statement, Anvisa and Roche said the full set of data available so far was not enough to meet the regulatory requirements needed to keep the conditional registration. With the cancellation, the import, distribution and sale of Elevidys in Brazil are officially suspended, reported Folha de S.Paulo.

Conditional registration is a provisional approval Anvisa grants to medicines for rare or severe diseases. The company must keep submitting data while the product is already in use; if the information fails to confirm the expected benefits, the registration can be canceled. Duchenne muscular dystrophy is a rare genetic disease that causes progressive muscle degeneration.

History of safety alerts

Elevidys obtained conditional registration in Brazil in December 2024. In July 2025, sales were suspended as a precaution after the therapy was linked to deaths from acute liver failure in the United States. According to Folha, the FDA, the U.S. regulator, linked three deaths to the gene therapy: two children with Duchenne muscular dystrophy who were using the medicine and one adult with limb-girdle muscular dystrophy.

In Brazil, ten children were treated with the drug before the suspension, and three reports of adverse effects had been received by Anvisa through July of last year, according to the agency. Elevidys was not incorporated into the SUS, Brazil's public health system, and reached patients through court orders. The Ministry of Health paid as much as R$ 17 million per dose, according to Folha.

Anvisa said the cancellation does not change Roche's obligations to monitor patients already exposed to the medicine, including the Brazilians who received Elevidys between December 2024 and July 2025, with continuous safety monitoring. Roche said it remains committed to the therapy's clinical program and to presenting new evidence, and Anvisa said it is willing to give priority review to future submissions of the medicine.