Zynteglo

2022: Announcement of Zynteglo

On August 17, 2022, the U.S. Food and Drug Administration (FDA) approved the gene therapy drug Bluebird Bio to treat a rare disease requiring regular blood transfusions. The drug company set the price for the drug at a record $2.8 million.

The approved drug is intended for the treatment of beta-thalassemia, which causes oxygen deficiency in the body and often leads to problems with the liver and heart. Patients with a severe form of the disease, of which there are about 1,500 in the United States, are forced to transfuse blood every two to five weeks.

Bluebird Bio launches world's most expensive drug

According to experts, the drug, which will be called Zynteglo, may face a certain disagreement of insurance firms due to its high price.

For example, Novartis in 2019 was forced to offer discounts and develop "results-based" installments for its $2.1 million Zolgensma drug after insurers refused to recognize the price of the drug.

Bluebird presents Zynteglo as a potential one-time treatment that could permanently eliminate the need for blood transfusions, resulting in cost savings for patients in the long term. According to Chief Operating Officer Tom Klima, the average lifetime cost of a blood transfusion could be $6.4 million.

The FDA warns of the potential risk of developing blood cancer from treatment, but notes that there have been no such cases in studies.

Bluebird expects to begin the process of treating patients in the fourth quarter of 2022. However, no therapy revenue is expected in 2022, as the treatment cycle will take an average of 70 to 90 days from initial cell collection to the final phase of treatment.^[1]

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