Medicine against SMA is no longer the most expensive in the world. Their record was broken by gene therapy Zynteglo worth $2.8 million.
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).