(Daily Caller) The U.S. Food and Drug Administration (FDA) on Wednesday approved a gene therapy for a rare blood disease which is set to reach the market at a record $2.8 million for a single dose, according to a press release by the therapy’s creator, Bluebird Bio.
Beta-thalassemia is an inherited blood disorder that causes a patient’s blood to fail to circulate oxygen through the body, according to the FDA press release concerning the approval. Bluebird’s new therapy, Zynteglo, infuses patients with cells that have a working copy of the gene responsible for the disorder, allowing the patient to produce blood that functions properly, according to a Bluebird press.
“[Zynteglo] has the potential to untether from the health care system patients who, on average, currently require transfusions every two to five weeks for life and may lose decades of life relative to the general population,” Bluebird Chief Communications Officer Sarah Alspach told the Daily Caller News Foundation. “We took this profound impact into consideration when pricing the therapy.”
Patients with beta-thalassemia typically require regular blood transfusions, which can lead to heart and liver problems when administered repeatedly, according to the FDA. Zynteglo is administered as a single dose therapy, and of 41 patients in clinical trials, 89% were able to forego transfusions, the FDA reported.
#NewsfromtheNest: We are thrilled to announce that the @US_FDA has approved our first gene therapy for adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions! Read more in our press release: https://t.co/eZwiJzNlGY [1/2] pic.twitter.com/prijXqSzft
— bluebird bio (@bluebirdbio) August 17, 2022