The U.S. Food and Drug Administration approved bluebird bio's Zynteglo (betibeglogene autotemcel), also known as beti-cel, for treating adult and pediatric patients with beta-thalassemia who need blood transfusions.
Zynteglo is a one-time treatment in which a patient's cells (bone marrow stem cells) will be genetically modified to produce beta-globin, an essential component of hemoglobin.
The approval was based on two Phase 3 studies, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and one long-term follow-up study, LTF-303. The Phase 3 studies included 41 patients with non-β0/β0 and β0/β0 genotypes, 89% of patients have achieved transfusion independence for at least 12 months, with weighted average hemoglobin of 9 g/dL. bluebird bio has announced that all the patients in the two Phase 3 trials were enrolled and treated.
bluebird has priced Zynteglo at $2.8 million (wholesale acquisition cost), and the company has announced that the price was based on robust clinical evidence. It further said that with Zyntelgo, complications because of transfusion and iron overload could be avoided. Additionally, the company stated that the lifetime transfusion costs nearly $6.4 million in the U.S. The company is also offering innovative contracts, including a single upfront payment and up to 80% risk-sharing.
Beta-thalassemia is an inherited genetic disorder where there will be a reduction in the blood cells and hemoglobin because of the mutation in the beta-globin subunit, resulting in an insufficient supply of oxygen to the body. In patients with transfusion-dependent beta-thalassemia, life-long blood transfusions were the standard of care. Because of the iron overload, there is a high risk of morbidity and mortality challenges.