Bluebird Bio gene therapy wins first FDA approval for rare blood disorder

A Bluebird Bio gene therapy that treats the rare blood disorder beta thalassemia is now approved by the FDA, providing the option for a one-time treatment that could eliminate the need for regular blood transfusions patients undergo as the current standard of care.

Somerville, Massachusetts-based Bluebird set a $2.8 million wholesale price for the product, Zynteglo, making it one of the most expensive therapies to reach the market. But the biotech said Wednesday that it has also come up with a reimbursement strategy that is tied to patients achieving benefit from the therapy.

Beta thalassemia is an inherited blood disorder that leads to lower levels of hemoglobin, the oxygen-carrying protein in red blood cells. The resulting low levels of oxygen in the body leads to dizziness, weakness, fatigue, and bone problems, among other complications.

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