Most expensive remedy in Brazil, of $ 11 million, is applied for the first time in SUS

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The SUS (Unified Health System) performed the first infusions of the high cost drug Elevidys (Delandistrogen MoxeparVoveque).

According to the Ministry of Health, two children with Duchenne muscle dystrophy received the drug this week at the Hospital de Clínicas de Porto Alegre (HCPA), a reference in the treatment of rare diseases.

Elevidys is the most expensive medicine in Brazil. The marketing value of each dose, as defined by the Medicines Market Regulation Chamber (CMED), was established at R $ 11 million.

With the acquisition and application of the drug, the Federal Government fulfills the Supreme Court (Supreme Court) order to manage Elevidys to patients who meet the criteria to receive it. The drug, developed to stabilize muscle function, albeit partially, is suitable for children aged 4 to 7 years.

Duchenne muscle dystrophy is a rare genetic disease that mainly affects male children. This genetic alteration is characterized by the lack or alteration of a protein in the muscle, dystrophin, which causes the main symptom of the disease: muscle weakness. The condition can lead to the progressive loss of motor skills such as running, jumping and climbing stairs.

Until then, the use of corticosteroids to slow the progression of muscle weakness was the only treatment available in the country.

Both patients will have medical follow -up and care to assess the effectiveness of therapy. The Ministry of Health states that the evidence available on Elevidys is still limited and that treatment has been debated by the scientific community.

The expectation, according to children’s neurologist Michelle Becker, responsible for the HCPA neuromuscular diseases, is to stabilize or make the disease progression slower.

“We don’t expect treatment to reverse that loss of strength that the patient has. It is very important to tell families that it is not a cure. So far, studies show that the drug is able to stabilize or delay the progression of the disease, but the duration of the effect is still limited, ”he said.

Families say they have hope

For Alessandro Neves, father of one of the children who received the infusion, the possibility of stabilization already brings hope.

“I don’t know if, in a moment, this medication can have a longer effect. There is still no evidence, but just stabilizing, we feel blessed. And, later, if you have any [medicamento] That is really the cure, we earn a little more time from this application, ”he said, according to material released by the Ministry of Health.

Luana Fassina, mother of the other child, also says he expects the drug to ensure a higher quality of life for his child.

“We were very well received, the team is wonderful and gave us all the support,” she said, according to the federal government.