'Only for millionaires': the USA has approved the most expensive medicine in the world - ForumDaily
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'Only for millionaires': in the United States approved the most expensive medicine in the world

Food and Drug Administration (FDA) approved the drug Zolgensma, the world's first gene-therapeutic agent for children under two years of age with spinal muscular atrophy (SMA), writes “Currently,".

Фото: Depositphotos

The drug corrects the damaged SMN1 gene that causes the disease and demonstrates impressive results, the regulator said in a statement.

Zolgensma is called the most expensive medicine in the world: the only infusion of the drug will cost $ 2,1 million, сообщил drug manufacturer, AveXis, a member of the Swiss pharmacological giant Novartis group.

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“Today’s approval marks another milestone in the transformative power of gene and cell therapies for a broad range of diseases,” said Ned Sharpless, acting FDA chief.

“We are on a path that we hope will one day lead us to the near eradication of SMA,” said Novartis head Vasa Narasimhan.

Zolgensma, or onasemnogene abeparvovec-xayoi, is a specially modified adenovirus that delivers and “writes” a copy of the correct SMN gene into motor neurons, the cells that control muscle movement.

The drug was approved on the basis of a special accelerated program that was guided by the results of an ongoing clinical trial: from 21 a child in the 19 test group did not simply survive (whereas without treatment, many children with SMA die because of respiratory failure) with AGR achievements: they can move their heads and sit without additional support. One research participant died and one ceased participation in the trials.

In another early clinical test, in the group of children who received an increased dose of Zolgensma, 75% of patients were able to sit without additional support, and 17% could stand up and walk.

Among the possible side effects of Zolgensma are acute liver damage, a decrease in the number of platelets in the blood, and vomiting.

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Zolgensma-like remedies for correcting problem genes are very expensive. Pharmaceutical companies claim that exorbitant prices are associated with a small number of patients: estimated Novartis, in the US, only about 1100 children can now get Zolgensma. This means that the costs of developing and testing the drug will be divided among themselves by a relatively small group of people.

Sometimes gene therapy drugs are made individually, which also affects the prices: for example, the first such means of gene therapy in the US Kymriah medicine for acute lymphoblastic leukemiaright after its approval cost $ 475000. Luxturna, a gene therapy treatment rare hereditary form of blindnesscosts $ 850 000.

Another gene therapy drug to combat SMA in patients of any age - Biogen's Spinraza can be even more expensive than Zolgensma, because it requires regular infusions. In the first year, treatment will cost $ 750 000, and then about $ 375 000 annually, all my life. Spinraza sales last year amounted to $ 1,7 billion.

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Novartis says that a one-time Zolgensma infusion will be cheaper for patients in the long term and offer 5-year installmentsas well as individual contracts that take into account the effectiveness of the funds in each case.

Critics point out the inadmissibility of such a price level for any kind of drugs and too much freedom of pharmaceutical companies in choosing the price of medicines for rare diseases.

“You can look at this from two sides. On the one hand, this is an amazing drug, only a few children need it, so think about it, a million here, a million there,” Peter Bach, head of the health policy center at Memorial Sloan Kettering Cancer Center, told STAT. - Or we have a big problem. Biopharma has completely pivoted to rare diseases because the market will tolerate any price and the FDA requires minimal data for approval.”

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