The Ministry of Health incorporated Zolgensma (onasemnogeno abeparvoveque), from Novartis Biosciences SA, for the treatment of children aged up to six months diagnosed with AME (Spinal Muscular Atrophy) type 1, considered the most serious.
The announcement was made by the Minister of Health, Marcelo Queiroga, on social networks. The ordinance (172 of December 6, 2022) with the decision was published this Wednesday (7), in the Official Gazette.
The medicine will be available in the SUS (Sistema Único de Saúde) within 180 days – the period necessary for the operational procedures of price negotiation, purchase, distribution and elaboration of a clinical protocol for guidance on use.
Zolgensma underwent an evaluation by Conitec (National Commission for the Incorporation of Technology in the SUS) and a public consultation — more than 1,200 participants sent contributions.
The therapy was approved in May 2019 by the FDA (Food and Drug Administration, a federal agency of the United States Department of Health and Human Services) and in August 2020 by Anvisa (National Health Surveillance Agency), with indication for children up to two years with SMA type 1.
The recommendation is that it be used in patients who are not on non-invasive ventilation for more than 16 hours a day.
SMA affects the spinal motor neuron and is caused by a change in the gene responsible for coding the protein necessary for proper muscle development. Rare, the disease causes weakness, hypotonia, atrophy and progressive muscle paralysis affecting breathing, swallowing, speech and the ability to walk. Type 1 affects 45% to 60% of all patients with SMA and can lead to death.
The treatment consists of applying a single dose in a hospital environment. The maximum price stipulated by the Medicines Market Regulation Chamber, the inter-ministerial body responsible for defining the maximum prices for the sale of medicines in the country, is R$ 6.4 million.
“Prioritizing the commitment to ensure access to Zolgensma, Novartis has aligned itself with the needs of the country, the Ministry of Health and patients to make the medication viable in a sustainable way in the SUS, through a value proposal that reflects its innovative character and is adequate to the Brazilian reality. Gene therapies represent a great advance, not only for patients and their families, but also for the entire health, science and technology system”, Novartis said in a note.
In December 2020, CMED had defined that a single dose of Zolgensma could not cost more than BRL 2.9 million in Brazil. The pharmaceutical questioned this value on the grounds that the drug is an innovative gene therapy that presents lasting results, while the competitor offers a continuous treatment.
The report asked the laboratory if there is a study on the production needed to meet the Ministry of Health. The pharmaceutical only said that “it is safely structured to guarantee full supply”.
THE Sheet asked the Ministry of Health about how many babies are eligible to receive the therapy, how often it will be purchased, how much it will cost the ministry and how Zolgensma will be dispensed for the application, but received no response.
Fátima Braga, president of Abrame (Brazilian Association of Spinal Amiotrophy), celebrates the incorporation of Zolgensma to the SUS. It is the third treatment available in the country — currently, Spinraza (nusinersena) and Evrysdi (risdiplam) are incorporated into the SUS for the treatment of AME. Both are in continuous use.
On the other hand, there are issues that need to be considered, according to Braga. “Not all Brazilian children have access to the heel prick test at birth. If we have gene therapy, we should have an early diagnosis of AME, which would change the course of the disease”, she says.
The association also questions the criterion of six months to live. “In some regions of the country, the diagnosis is late, it can take up to a year. There is still a history of mechanical ventilation. Often, children with AME 1 or zero cannot breathe on their own. There are children who spend 24 hours with the equipment “, says the president of the entity.
She also highlighted the risk sharing —the associations were not informed about the criteria— and the application costs as dark points.
According to Braga, only São Paulo and Rio Grande do Sul are applying. “The transfer must be done in a private plane. The child needs to be observed for a month after the application. Where will the families stay? In a hotel? Who will bear these costs? In Ceará, two patients who received the therapy had to go to Albert Einstein”, concludes Braga.
Zolgensma should also be included in the ANS (National Supplementary Health Agency) list and be covered by health plans within 180 days.
By Law 14.307/2022, which defines rules for the incorporation of new treatments by health plans and insurance, all technologies evaluated and recommended by Conitec for SUS must also be incorporated into supplementary health within the same period.
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