Bluebird Bio's gene therapy beti-cel receives FDA priority review

CompilationTom Lee

A few days ago, the U.

The FDA's priority review decision to accept the therapy was based on data from two Phase 3 studies of the therapy, Northstar-2 and Northstar-3, and a long-term follow-up study, LTF-303

Beta-thalassemia is a serious genetic disorder in which patients have genetic mutations that lead to significantly lower levels of hemoglobin, leading to severe anemia and lifelong dependence on blood transfusions

Anne Virginie Eggimann, chief regulatory officer of Bluebird Bio, said that for a long time, beta-thalassemia patients who depended on regular blood transfusions had to endure a huge burden associated with the disease

Regulatory filing for the gene therapy beti-cel has been a long road for Massachusetts-based Bluebird Bio, which reported its Phase 3 data in June 2017 and received it in Europe two years later approved

In Germany, the only country that has released pricing details, local regulators have offered to pay $790,000 per patient, which would rise to $950,000 after five years if the treatment was still effective

Reference source: Bluebird bio's beti-cel accepted for priority review by FDA