City of PI is experiencing rare disease boom and medicine costs R$ 199 thousand

According to her, even with underestimated numbers, Brazil is the country with the second highest number of cases of this disease in the world, behind only the USA. Patients’ hope increased in April, when Anvisa granted registration of the first medicine to treat ataxia in the country, the skyclarys (omaveloxolone), which slows the progression of the disease.

Amália is the mother of a person with ataxia who has been taking this medication for two years and lives outside Brazil. “He was the first participant in the study in the US and still takes it today.” “His numbers in the neurological evaluation are almost the same as they were eight years ago,” he says.

“We are already behind in relation to the United States. In the case of Brazil, those who have money go there and buy, but on our list, there is only one person I know who is able to buy. The others, no.”

Amália explains that the pharmacist did not submit the request to incorporate the medicine into the SUS. “She argues that, over the last year, the government has rejected requests to incorporate medicines for rare diseases. The government has also not expressed interest in purchasing, and we are at this impasse”, he says.

With no possibility through the SUS, patients are seeking justice to gain access. According to the column, there is a patient, from Pernambuco, who is already receiving the drug. “We still have three other actions asking for the skyclarysand one already with a favorable decision, but still awaiting implementation”, says Priscilla Mourão, the lawyer responsible for the country’s first requests.