Over 3 million euros to treat a rare form of atrophy that affects children
3 million and 600 thousand euros (about 4 million francs) is the cost of the most expensive drug in the world. Presented last week by the pharmaceutical company Novartis, the drug is used to treat type 1 spinal muscular atrophy and will go on sale over the next year.
The drug is called AVSX-101 and it is a gene therapy that promises a single infusion to have positive effects on patients, mostly children who are born with this form of atrophy. Commonly known as SMA, it is a very serious neurological pathology that prevents the young patients who are affected by it to breathe or swallow autonomously but also to crawl, walk and control head and neck movements.
What is SMA?
The SMA it is a disease that occurs only if both parents are carriers of the gene responsible for the disease. According to data, children affected by this rare form of atrophy cannot survive the second year of life.
The recently developed drug injects a gene capable of stopping the muscle degeneration through modified viruses. According to a first clinical test carried out on fifteen children, the treatment seems to work so much that they have passed the two years of life and some even managed to walk.
Meanwhile, the controversy is raging over the excessive cost of the drug, even though they let the company know that the cost is justified "with a gain of at least thirteen years of life".
