Roche Pulls Elevidys Registration in Brazil After Anvisa Suspension

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PHARMA · NEUROSCIENCE · SEPTEMBER 10, 2026 · LATIN AMERICA

Brazil’s most expensive approved drug no longer has a registration. On August 24, 2026, Anvisa, Brazil’s national health surveillance agency, cancelled the conditional registration of Elevidys (delandistrogeno moxeparvoveque) — a gene therapy for Duchenne muscular dystrophy (DMD) — at the explicit request of its manufacturer, Roche, according to the agency’s announcement published in the Diário Oficial da União. The cancellation formalises what a July 2025 precautionary suspension had already signalled: the evidentiary case for the therapy in Brazil had collapsed before it could be built.

The mechanism matters. Brazil’s conditional registration is a provisional pathway, granted by Anvisa for medicines treating rare or serious diseases, that permits commercialisation while the manufacturer continues submitting confirmatory data. It is not a permanent approval; it is a regulatory wager that post-market evidence will arrive in time to satisfy the agency’s requirements. In Elevidys’s case, that wager failed. Anvisa stated that ‘the totality of available data for Elevidys was not sufficient to meet the regulatory requirements in force necessary to maintain the conditional registration,’ per the agency’s cancellation notice. Roche, rather than contest the finding, requested the cancellation itself.

A price record with no commercial future

The drug had carried the highest approved price ever set for a medicine in Brazil. The Câmara de Regulação do Mercado de Medicamentos (CMED), Brazil’s drug-pricing regulator, established a commercialisation price of R$13.6 million per dose; the price for the Sistema Único de Saúde (SUS), Brazil’s public health system, was set at R$10.6 million, according to Valor Econômico. Folha de S.Paulo reported the cost per dose reaching up to R$20 million depending on state-level tax incidence, while the Ministério da Saúde paid R$17 million per dose in purchases made under court orders. For context, Denizar Vianna, a former secretary of science and technology at the Ministério da Saúde, told Valor Econômico in January 2025 that at R$17 million per dose and approximately 300 eligible patients, Elevidys would have generated an annual public and private health impact of R$3.6 billion — a figure he compared to the R$1.2 billion hemophilia programme budget that served 32,000 people in 2023.

Those figures are now moot for new patients. With the cancellation, the importation, distribution, and commercialisation of Elevidys in Brazil are officially suspended, per Anvisa’s notice. The ten children treated in Brazil between December 2024 and July 2025 — the window between conditional registration and the precautionary suspension — remain subject to Roche’s post-market monitoring obligations. Anvisa was explicit that the cancellation does not extinguish those obligations, a distinction with practical consequence: Roche must continue tracking adverse events and clinical outcomes for the exposed cohort regardless of its commercial exit.

Three adverse-event notifications had been received by Anvisa before the July 2025 suspension, according to Folha de S.Paulo. In the United States, the Food and Drug Administration (FDA) had linked three deaths from acute liver failure to the same gene therapy, a safety signal that Folha reported was associated with the Brazilian suspension decision. The FDA’s findings are a separate regulatory matter and have not been adjudicated as a final causal determination, but they formed part of the evidentiary context Anvisa was evaluating.

Judicialization as the access channel — and its limits

Elevidys reached most Brazilian patients not through the SUS formulary but through court orders. In 2024, approximately 70 lawsuits sought coverage at the US price of US$2.8 million per dose — equivalent to R$17 million — generating a potential cumulative liability to the federal government of R$1.2 billion, according to Valor Econômico. The Advocacia-Geral da União (AGU), Brazil’s federal legal office, estimated that if all 55 active judicial actions at the time had been granted, the cost to the public health system would have reached R$1.15 billion, per AGU’s own figures reported by Valor. The Supremo Tribunal Federal (STF), Brazil’s supreme court, became the venue for managing this pressure: Justice Gilmar Mendes suspended a portion of the injunctions in August 2024, opened a conciliation process between the federal government and Roche, and ultimately oversaw a settlement homologated in December 2024 that allowed the Ministério da Saúde to purchase doses for patients meeting clinical criteria.

By January 2025, the Ministério da Saúde had acquired doses for two patients, delivered through the Hospital de Clínicas de Porto Alegre (HCPA), a reference centre for rare diseases. A third patient received the therapy at the Hospital Fernandes Figueiras in Rio de Janeiro, according to Folha de S.Paulo’s reporting on the STF conciliation update in February 2025. The STF formally concluded its conciliation work on February 26, 2025 — five months before the commercial suspension and eighteen months before the registration cancellation.

The judicialization pathway is now closed by the cancellation itself. Justice Mendes had previously barred any judicial grant of Elevidys outside the terms of Anvisa’s registration; with no registration in force, there is no regulatory framework within which a court could order supply. Families of children with DMD who had not yet received treatment under existing injunctions face an access gap with no immediate legal remedy, a situation the available record does not yet clarify in terms of pending litigation outcomes.

The principal counterargument to reading this cancellation as a definitive regulatory failure is that Roche initiated the request — which could reflect a strategic calculation rather than a concession of defeat. Both Roche and Anvisa stated publicly that the agency ‘reaffirms its willingness to evaluate on a priority basis future submissions that bring additional evidence, subject to current regulatory requirements,’ per the cancellation notice. Roche, for its part, stated it ‘remains committed to the clinical programme and to presenting the necessary evidence.’ One possible scenario is that Roche is managing the conditional registration’s evidentiary clock — exiting a framework it could not satisfy on the current data timeline while preserving the option to re-file once a more complete dataset is available. Whether that re-submission materialises, and on what timeline, has not been publicly disclosed.

For the broader gene therapy market in Brazil, the episode tests the conditional registration pathway in a way no prior case had. Novartis’s Zolgensma, a gene therapy for spinal muscular atrophy (SMA) and the previous holder of Brazil’s highest drug price, navigated a risk-sharing agreement with the Ministério da Saúde that tied payment to patient outcomes — a model the government had sought to replicate for Elevidys before the safety suspension intervened. The Zolgensma precedent now stands as the reference for how a gene therapy can sustain a conditional registration in Brazil; Elevidys’s trajectory is the counter-example. No analyst estimate of the impact on Roche’s Brazilian revenue has been published in connection with this cancellation, though Valor Econômico reported in January 2025 that Roche’s total Brazilian pharmaceutical revenue was R$4.6 billion annually, with innovative products representing more than half of that figure.

The deeper signal is structural: Brazil’s conditional registration pathway was designed to accelerate access for patients with rare diseases while evidence accumulates — but the Elevidys case demonstrates that the pathway also creates a defined exit mechanism when that evidence does not arrive. For manufacturers, the implication is that a conditional registration in Brazil is not a durable commercial position; it is a time-limited licence whose continuation depends on data delivery, not on market presence alone.