Beta thalassemia is a rare genetic blood disorder characterized by a substantial reduction in or lack of β-globin protein and resulting in chronic anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. No curative therapy exists for beta thalassemia other than an allogenic hematopoietic stem-cell transplantation (HSCT) with a matched (most often sibling) donor. 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, substantially affecting their quality of life (QOL). A high unmet exists for therapies to reduce or eliminate the burden of transfusion. Drug developers have recognized the commercial opportunity in the beta thalassemia market and have focused on developing agents with novel mechanisms of action that target the underlying genetic defect and reduce the transfusion burden.
- How large is the diagnosed prevalent beta thalassemia population in the United States and EU5?
- How will the population change through 2027? What is the current treatment landscape for beta thalassemia patients, and how will it change in the next ten years?
- What clinical needs remain unfulfilled?
- 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.