By Mrinalika Roy
(Reuters) -Bluebird bio on Wednesday won U.S. approval for its gene therapy to treat a rare blood disorder, a treatment that is set to become the expensive on record with a price of $2.8 million.
The Food and Drug Administration’s approval allows the use of beti-cel, to be branded as Zynteglo, as a one-time treatment for pediatric and adult beta-thalassemia patients dependent on regular transfusions.
Beta-thalassemia is an inherited disorder caused by beta-globin gene mutations that reduce the production of hemoglobin. Treatment for people with the disorder often consists of lifelong regimens of chronic blood transfusions.
Bluebird is positioning its therapy as a potential cure for transfusion dependent beta-thalassemia patients.
Gene therapies have faced insurance-related hurdles in the past due to their high costs. Bluebird has tried navigate the issue by entering talks with several insurance companies, most of which favor a one-time payment option.
“We’ve been speaking with payers about a one-time upfront payment. Potentially, up to 80% of that payment will be reimbursed if a patient does not achieve transfusion independence, they’re very excited about that,” Chief Operating Officer Tom Klima said prior to the approval.
Beti-cel works by adding functional copies of a modified form of beta-globin in a patient’s stem cells in order to correct the hemoglobin deficiency.
Bluebird plans to launch the product this year and expects to start treatment process for patients in the fourth quarter.
The company, however, does not expect any revenue from the therapy in 2022 as the treatment cycle would take an average 70 to 90 days from initial cell collection to final transfusion.
(Reporting by Mrinalika Roy in Bengaluru; Editing by Aditya Soni)
