Injection for $2.8 million

The most expensive medicine in the world has become a new therapy for beta-thalassemia

Polina Loseva, N+1

In the USA, gene therapy for beta-thalassemia, a hereditary blood disease, has been approved. The developer of the drug, Bluebird bio, claims that one injection is enough to, if not cure, then significantly improve the patient's condition. But such an injection will cost $2.8 million. This is 700 thousand dollars more expensive than the injection of "Zolgensma" — the previous holder of the title "the most expensive medicine in the world".

Until recently, Zolgensma, a medicine for spinal muscular atrophy, was considered the record holder among expensive medicines. Treatment for her consists of a single injection, which supplies the body with a healthy copy of the gene and should prevent the development of the disease. But this injection itself costs a little more than two million dollars.

Now "Zolgensma" has gone down to second place, and the first was taken by a new drug, "Zynteglo" (Zynteglo) from Bluebird bio. It has been approved by the U.S. Food and Drug Administration (FDA) as a treatment for beta-thalassemia. The cost of such treatment will be 2.8 million dollars.

Beta-thalassemia is a hereditary disease in which one of the chains of hemoglobin in red blood cells is little or no. Depending on the specific mutation, pathology can manifest itself in different ways: from mild weakness and pallor to serious problems when a person constantly needs transfusions of donated blood.

"Zinteglo" works like a classic ex vivo gene therapy: the hematopoietic cells of the bone marrow are taken from the patient, treated with viral vectors with the desired hemoglobin gene, and then returned back to the body. The technology assumes that the cells will take root immediately, and therefore it is enough to perform the procedure once. At least in clinical trials, according to the company in a press release, 32 of the 36 participants became completely independent of blood transfusions - that is, their hematopoietic cells learned to produce enough hemoglobin.

The manufacturers of Zolgensma, the previous record-holder drug, justified the high price of the drug something like this: its closest competitor, Spinraza gene therapy, requires repeated administration, and also costs a lot — several hundred thousand a year. Therefore, in a few years, the cost of a single injection (if, of course, it turns out to be effective) should pay off.

The creators of "Zinteglo" are guided by approximately the same logic: patients with severe beta-thalassemia need a blood transfusion once every two weeks, and for a lifetime (which often turns out to be shorter than average) they spend more than six million dollars on treatment. Against the background of this figure, 2.8 million does not seem to be such an impressive amount anymore.

However, it will not work at all without doctors after a single injection of "Zinteglo". Manufacturers warn: after gene therapy, there is a risk that blood cancer will develop. Therefore, those who have received a new hemoglobin gene are advised to undergo an examination annually at least for the first 15 years after treatment, and ideally — until the end of life.

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