Bluebird’s Zynteglo Approval Kicks Off Commercial Operations
Beta-Thalassemia Gene Therapy’s List Price Is $2.8m
Bluebird bio’s Tom Klima told Scrip launch preparations are well under way, including negotiations with payers on outcomes-based reimbursement agreements.
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The drug pricing watchdog said improvement to patients’ quality and length of life from lovo-cel and exa-cel were likely, but noted ongoing uncertainty around durability and toxicity concerns.
Data presented at ASH show patients maintaining transfusion independence for years after infusion, months after bluebird won FDA approval for the beta-thalassemia gene therapy.
Early data in Gaucher disease suggest Avrobio’s hematopoietic stem cell gene therapy could help patients who do not response to enzyme replacement therapy.