After US FDA Approval, Bluebird’s $2.8M gene therapy becomes the most expensive drug

The US Food and  Drug Administration approved Bluebird Bio’s gene therapy for patients with a rare disorder needing regular blood transfusions, and the drugmaker priced it at a record $2.8 million.

The approval sent the pharmaceutical firm’s shares 8% higher.The drug is for the treatment of beta-thalassemia, which causes an oxygen shortage in the body and often leads to heart and liver problems.

The sickest patients, estimated to be equal to 1,500 in the United States alone, require blood transfusions every two to five weeks.

Analysts explained that the therapy, to be rebranded as Zynteglo, is expected to face some resistance from insurers due to its steep price.

Gene therapies generally come with a high price tag as they are often curative and have faced hurdles in securing insurance coverage.

For example, in 2019, Novartis was forced to offer discounts and work out “outcome-based” installment payments for its $2.1 million therapy after insurers flinched at the drug’s price.

Bluebird has pitched Zyntelgo as a possible one-time treatment that could solve the need for transfusions, resulting in savings for patients over the long term.

According to Chief Operating Officer of Bluebird Bio, Tom Klima, the average cost of transfusions over the lifetime can be $6.4 million. He felt the firm’s price still brings a significant value to the patients.