Find out which drugs used in rare diseases are highly regarded on the market
Medicines are essential for treating various health problems. Some are also extremely expensive. In general, this happens with those used by a small portion of the population suffering from some type of rare disease, affecting 65 people per 100,000, according to the WHO (World Health Organization).
According to information from the Ministry of Health, about 13 million Brazilians have some kind of rare disease. As a result, the price of some drugs is overrated in the market, as the fewer people use a drug, the greater its commercial value.
Below, check out a list of the world’s most expensive drugs marketed in Brazil.
1. Zolgensma – $ 2.1 million
Indicated for the treatment of Spinal Muscular Atrophy (SMA), the medicinal It is considered the most expensive in the world. This is because it uses a high investment technique to be produced, as well as being developed and marketed exclusively by the pharmaceutical company ‘Novartis’.
According to information from Anvisa (National Health Surveillance Agency), drug treatment is indicated for children up to 2 years of age. Furthermore, it can counteract the effects of muscle atrophy. Regarding the dose of application, the agency informs that it must be performed by a specialized professional according to the weight of each patient.
Two. Luxturna – 850 thousand dollars
Used in the treatment of patients with progressive vision loss caused by hereditary dystrophy, the drug administered by the subretinal route is indicated for pediatric patients over 4 years of age. Also, it should only be used in surgical environment, according to information from Anvisa. Despite the advantages, it is not yet offered by the SUS.
3. Ravicti – $ 793,000
Manufactured by “Horizon at your side”, the drug is indicated in the treatment of urea cycle disorders. It is responsible for preventing the accumulation of ammonia in the body, caused by the failure to eliminate the waste chemical element nitrogen, which is harmful to the health of the body. According to information from the European Medicines Agency, “Ravicti” is used when diseases cannot be cured by diet alone. It must be administered orally and obtained by prescription.
4. Brineura – 700 thousand dollars
According to the information contained in the package insert of the drug, it is indicated for patients suffering from type 2 neuronal ceroid lipofuscinosis, also known as “tripeptidylpeptidase deficiency”, a disease that constitutes a group of neurodegenerative diseasesdue to a deficiency of the enzyme called tripeptidyl peptidase 1. The drug should be administered by a doctor in a hospital or clinic and according to the age of the patient.
5. Carbaglu – US $ 419 thousand
According to the drug registration at Anvisa, ‘Carbaglu’ is used in the treatment of hyperammonemia, a metabolic disorder caused by N-acetylglutamate synthase (NAGS) deficiency, which affects the body and impairs the development of children and adults at any age. . In the long term, drug intervention can reduce neurological complications caused by the accumulation of ammonia in the bloodstream.
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Source: Terra
Benjamin Smith is a fashion journalist and author at Gossipify, known for his coverage of the latest fashion trends and industry insights. He writes about clothing, shoes, accessories, and runway shows, providing in-depth analysis and unique perspectives. He’s respected for his ability to spot emerging designers and trends, and for providing practical fashion advice to readers.
