Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein, which results in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. Allogenic hematopoietic stem cell transplantation with a matched (most often sibling) donor is the only available curative therapy; however, with the recent approval of bluebird bio’s Zynteglo in Europe and expected commercial launch starting in 2020, patients will soon have an additional potentially curative option. Most patients are currently managed with a combination of blood transfusions and iron chelators. The most severe thalassemia major patients require chronic blood transfusions to survive, which substantially impacts quality of life. A high unmet need exists for therapies to reduce or eliminate transfusion burden. Drug developers have recognized the commercial opportunity in the beta thalassemia market, and have focused on developing agents that target the underlying genetic defect and reduce transfusion burden.
- How large is the diagnosed prevalent beta thalassemia population in the United States and EU5? How will the population change through 2028?
- What is the current treatment landscape for beta thalassemia patients, and how will it change in the next ten years? How will the launches of Zynteglo and luspatercept impact beta thalassemia treatment?
- What pipeline products are most promising, and what sales/uptake could they secure in beta thalassemia? How will new therapies affect medical practice?
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.