FDA Approves Gene Therapy, Manufacturer Prices Drug at Record $2.8 Million

After the FDA approved Zynteglo (betibeglogene autotemcel) on Aug. 17, the gene therapy’s manufacturer, bluebird bio, Inc., announced the drug would have a $2.8 million wholesale acquisition cost (WAC). That is the highest WAC for a drug hitting the market in the U.S.

Zynteglo is indicated as a potential cure for patients with transfusion-dependent beta-thalassemia, a rare disorder caused by genetic mutations in cells that carry oxygen throughout the body. Such patients typically require red blood cell transfusions every two to five weeks.

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