27 May 2019

The most expensive

In the USA, the regulator approved the most expensive medicine in the history of mankind


In the US, regulators approved a drug worth $2.125 million. The most expensive drug in history was created to combat a rare disorder that destroys muscle control and kills almost all patients in a few years.

The drug Zolgensma from the Swiss manufacturer Novartis is a gene therapy for spinal muscular atrophy. The terrible disease weakens the child's muscles so much that he cannot move, and then swallow and breathe. This is reported by the press release of the FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality.

Every year in the United States, the disease affects about 400 children. The medicine is intended for children under the age of two and is intended for patients with any of the four types of terrible disease. The therapy consists of a one-time infusion, lasting about an hour.

If the treatment does not work, the manufacturer is ready to provide a partial discount, and will also allow insurers to pay its cost for five years ($ 425 thousand annually).

Another cure for this disease is Spinraza. Its cost is $ 750 thousand in the first year and then $ 350 thousand the next. However, the medicine allowed in the United States should be taken every four months, not once.

Note that muscular atrophy of the spine is caused by a defective gene that does not allow the body to produce enough protein for the nerves that control movement. As a result, they are rapidly dying off.

Zolgensma provides a healthy copy of the defective gene. Thanks to this, the necessary protein is produced, and the child can develop normally.

It is too early to say whether the medicine cures this disease forever. However, children who received the medicine in the first six months of life have no symptoms of the disease, although they are already 5-6 years old.

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