Americans have created the most expensive drug worth $2.8 million. What is Zynteglo?

The SMA drug, Zolengsma, is no longer the most expensive in the world. His record was broken by the $2.8 million gene drug Zynteglo .

On August 17, the FDA approved a gene therapy from the American company Bluebird Bio, designed to treat a rare disorder – beta thalassemia, which leads to a decrease in hemoglobin and, as a result, a lack of oxygen in the body. Chronic SpO2 deficiency causes heart and liver problems. The most severe patients with this disorder require regular blood transfusions every 2 to 5 weeks. There are about 1500 of them in the USA.

Bluebird markets Zynteglo as a one-time treatment. Patients who receive it will no longer need a transfusion, so this will result in savings in the long run. The average lifetime cost of a transfusion can be as high as $6.4 million.

Bluebird expects to start treating the first patients at the end of 2022. The treatment cycle will last from 70 to 90 days – from the initial collection of cells to the introduction of the final dose of the drug. Each dose of Zynteglo will be custom made from the patient’s own cells (bone marrow stem cells) that have been genetically engineered to produce functional beta-globin (a component of hemoglobin).