Those with deep pockets and a rare disease, beta-thalassemia, often requiring life-long blood transfusions, have something to rejoice about. On Wednesday, the U.S. Food and Drug Administration approved bluebird bio’s (BLUE.O) gene therapy, Zynteglo, as a one-off treatment and priced it at a record $2.8 million. Beta-thalassemia causes an oxygen shortage in the body and often leads to liver and heart issues. As reported by Channel News Asia, the sickest patients, estimated to be up to 1,500 in the United States, need blood transfusions every two to five weeks. In contrast, Bluebird’s Zynteglo will potentially be a one-time treatment that will do away with the need for transfusions.
Even the cost of transfusions isn’t economical, with its average cost (over a lifetime) totaling nearly $6.4 million. Chief operating officer Tom Klima told Reuters before the approval. “We feel the prices we are considering still bring a significant value to patients. Potentially, up to 80 per cent of that payment will be reimbursed if a patient does not achieve transfusion independence, they (insurers) are very excited about that,” The therapy is expected to commence towards the end of 2022. The treatment cycle would take an average of 70 to 90 days from initial cell collection to final transfusion.
At $2.8 million, this one-time gene therapy has become the most expensive medicine in history
