Medicine against SMA is no longer the most expensive in the world. Their record was broken by gene therapy Zynteglo worth $2.8 million.
August 17 FDA approved gene therapy from of the Bluebird Bio company, intended for the treatment of a rare disorder – beta-thalassemia, which causes a decrease in hemoglobin and, as a result, a lack of oxygen in the body. In the future, this often leads to problems with the heart and liver. The most severe patients with this disorder require regular blood transfusions every 2-5 weeks. There are about 1,500 of them in the US.
Bluebird introduced Zynteglo as a single treatment. Patients who receive it will no longer need transfusions, so this will save money in the long run. The average cost of a transfusion over a lifetime can reach $6.4 million.
Bluebird expects to start treating the first patients in late 2022. The treatment cycle will last 70 to 90 days, from the initial collection of cells to the final transfusion. Each dose of Zynteglo is a personalized treatment created using the patient’s own cells (bone marrow stem cells) that have been genetically modified to produce functional beta-globin (a component of hemoglobin).