A shot for three million

The new most expensive medicine in the world has become a drug for cerebral adrenoleukodystrophy

Anastasia Lyashenko, N+1

Bluebird bio company from the USA has set a new record for the cost of the drug, bringing to the market a drug for cerebral adrenoleukodystrophy (a rare brain disease in children), which costs $ 3 million. In April of the same year, Bluebird bio introduced another drug, which at that time also won the title of the most expensive in the world — $ 2.8 million for one injection.

The previous development of Bluebird bio was supposed to help patients with a hereditary blood disease — beta-thalassemia. This disease is associated with a small amount or complete absence of one of the hemoglobin chains, and their drug, Zinteglo, approved by the US Food and Drug Administration (FDA), allowed patients to avoid constant blood transfusions with a single injection.

The new drug, Skysona, will be the first approved drug for the treatment of cerebral adrenoleukodystrophy, a rare brain disease that occurs in young boys and leads to disability or death. In the USA, about five children with this disease are born every year, Bluebird bio expects to annually produce a drug for ten children from 4 to 17 years old.

Cerebral adrenoleukodystrophy is a hereditary neurometabolic disease that occurs due to a mutation of the transmembrane transporter protein Adrenoleukodystrophy Protein, which disrupts the transport of very long—chain fatty acids. The disease is characterized by demyelination of the central nervous system and spinal cord, as well as adrenal insufficiency.

The use of sibling stem cells can slow down or stop the development of the disease, but most patients do not have them. Skysona can help just such children by changing the gene responsible for cerebral adrenoleukodystrophy.

In the main testing, 29 out of 32 patients did not develop functional disorders in two years, but three patients were diagnosed with bone marrow cancer, and the FDA is concerned about how many more patients may face such a disease after some time. On the other hand, this type of cancer is treatable and two out of three patients have already gone into remission.

The FDA still has questions about the analysis of data on the tests performed and to what extent the benefits outweigh the potential risks for patients. At the moment, "Skysona" is the best solution for boys who do not have siblings for transplantation. Bluebird bio expects that the drug will become available at the end of this year.

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