Brazilian public hospitals now offer BRL 7 million drug for free

Brazil’s Ministry of Health on Thursday (May 15) launched the first Zolgensma treatments in the country’s national health-care network, the SUS. The drug—one of the most expensive in the world, which can cost around BRL 7 million from private hospitals—is used to treat type-1 spinal muscular atrophy (SMA), and was recently incorporated into the SUS.
The drug was first istered simultaneously in Brasília and Recife. In the country’s capital, Health Minister Alexandre Padilha went to the José Alencar Children’s Hospital to monitor the case of a baby diagnosed with type-1 SMA.
“Brazil is one of only six countries to offer this extremely innovative medication, which is expensive for families. Treatment usually ranges from BRL 7 million to BRL 11 million per dose. It would be impossible for families to bear this cost,” he said.
The move came through a deal signed with the international industry that makes payment conditional on the outcome of the therapy in the patient. Before type-1 SMA technologies were available in the SUS, children with the disease had a high probability of dying by the age of two.
Three new requests for the drug have been made to SUS patients, the minister reported, who will be included in a protocol for tests and treatment. This protocol establishes a specific flow for the treatment of the disease in public hospitals. The minister estimates that more than 100 patients with a recommendation to use the drug will be treated over the next few years.
This type of therapy is recommended for patients up to six months old who are not on invasive mechanical ventilation for more than 16 hours a day. According to Brazil’s Ministry of Health, with the incorporation of Zolgensma, the SUS will now offer all disease-modifying therapies for type-1 SMA. To start treatment for spinal muscular atrophy, a patient’s family must one of the 28 gene-therapy centers available in 18 states.
Of the 2.8 million Brazilians born alive in 2023, 287 were diagnosed with the disease, as per official data. The treatment uses medication to replace the function of a gene that is missing or not working properly. The disease is rare and affects body movements as well as breathing.