Medication will be offered to babies up to 6 months with type 1 spinal muscle atrophy; Payment The laboratory is conditioned to results
Health announced on Thursday (20.mar.2025) the incorporation of zolgensma into the SUS (Unified Health System), a drug for children with AME (spinal muscle atrophy) type 1.
AME Type 1 is a neuromuscular disease that leads to progressive child muscle atrophy. This is the first time that a gene therapy (insertion of genes in cells and tissues) is available free of charge in the Brazilian public network.
Brazil should join a small group of 6 countries that offer this medicine in their public health systems. The treatment, one of the most expensive in the world, has an average cost of $ 7 million per patient.
According to the ministry, AME affects 65 people in every 100,000 individuals. Treatment will be offered to children up to 6 months old who are not in invasive mechanical ventilation for more than 16 hours a day.
The agreement between the Ministry of Health and Novartis, manufacturer of Zolgensma, introduces a risk sharing model. Payment is conditioned to the therapeutic results observed in patients.
For the implementation of therapy, the Ministry of Health and Novartis are preparing the qualification of teams in 28 reference services for the treatment of AME in SUS, distributed in 18 Brazilian states.
The monitoring of patients treated with zolgensma will be performed by a permanent committee, with results monitored for 5 years.
