Beta thalassemia (BT) is a rare genetic disorder characterized by the reduced production of hemoglobin. It is caused by a mutation in one hemoglobin beta (HBB) gene (beta thalassemia minor) or a mutation in both HBB genes (beta thalassemia major; some patients with mutations in both genes can be classified as beta thalassemia intermedia). The mutation can cause jaundice, failure to thrive, and chronic anemia. Management of BT was once limited to regular blood transfusions and iron chelators; however, more-recent therapies such as Bristol Myers Squibb / Acceleron’s Reblozyl and Bluebird Bio’s Zynteglo offer a more-promising future. Reblozyl, an erythropoietin maturation therapy, launched in the United States in late 2019 and in Europe beginning in 2020 to treat anemia in adult patients. Zynteglo, a LentiGlobin vector-based gene therapy, is expected to launch in the United States in 2022; the therapy was available in Europe but was withdrawn by Bluebird Bio because of unfavorable pricing negotiations. Strong unmet need remains for universally curative therapies that improve patients’ quality of life. Drug developers are focusing on agents that target the underlying genetic defect and reduce the transfusion burden.
United States, EU5
Six country-specific interviews with thought-leading hematologists
Supported by survey data collected for this study
Diagnosed prevalent and drug-treatable cases of beta thalassemia by country, segmented by severity, genotype, and availability of HLA-matched sibling HSCT donor
Drug-level sales and patient shares of key beta thalassemia therapies through 2031, including Reblozyl and Zynteglo
Phase II: 6 drugs; coverage of select early-phase products
Marketed drugs: 2
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