The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million.
The approval sent the company’s shares 8 percent higher and is for the treatment of beta-thalassemia, which causes an oxygen shortage in the body and often leads to liver and heart issues.
The sickest patients, estimated to be up to 1,500 in the United States, need blood transfusions every two to five weeks.
The therapy, to be branded as Zynteglo, is expected to face some resistance from insurers due to its steep price, analysts say….