Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of beta-globin protein, resulting in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. Until 2019, allogenic hematopoietic stem cell transplantation (HSCT) with a matched (most often sibling) donor was the only curative therapy. However, with the launch of Bluebird Bio’s Zynteglo, some beta thalassemia patients have the possibility of a transfusion-free future. Zynteglo is a lentiglobin vector-based gene therapy that launched in Europe for transfusion-dependent patients aged 12 or older who do not have a β0/β0 genotype and for whom HSCT is appropriate but cannot be administered owing to a lack of an HLA-matched related donor. Luspatercept is another treatment for beta thalassemia; it was launched in 2019 in the United States and in 2020 in Europe to treat the anemia in adult beta thalassemia patients who require regular red blood cell transfusions. Most patients are managed with a combination of blood transfusions and iron chelators. Patients with the most severe form of the disease require chronic blood transfusions to survive, substantially affecting their quality of life. Thus, therapies that reduce or eliminate the transfusion burden are greatly needed. Recognizing the commercial opportunity in this market, drug developers have focused on agents targeting the underlying genetic defect and reducing 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, age cohort, genotype, and availability of HLA-matched sibling HSCT donor.
Drug-level sales and patient shares of key beta thalassemia therapies through 2030, including Zynteglo and luspatercept .
Phase II and Phase I/II : 6 drugs. Coverage of select preclinical and Phase I products.
Marketed drugs: 2
Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.