Brasília – The Ministry of Health, through the Secretariat for Science, Technology, Innovation and the Health Industrial Complex (SECTICS), has launched a public consultation to evaluate the expansion of Zolgensma’s therapeutic use within the Unified Health System (SUS).

Currently, the gene therapy is available only for patients diagnosed with spinal muscular atrophy (SMA) type 1. The proposal now under discussion would extend access to children under two years of age who have biallelic mutations in the SMN1 gene and up to three copies of the SMN2 gene, following criteria aligned with recent court decisions.

Zolgensma was incorporated into the SUS through a risk-sharing agreement between the Ministry of Health and Novartis, at a cost of R$ 5.7 million per dose, making it one of the world’s most expensive treatments. Since May 2025, the therapy has been provided to children with SMA type 1, with an initial goal of treating 137 patients in the first two years.

The public consultation will remain open until October 20, 2025, through the Participa + Brasil platform.

Source: Futuro da Saúde

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