Beta thalassemia is a recessive monogenic blood disorder characterized by low levels or an absence of the protein ß-globin, a key component of the oxygen transporting protein hemoglobin. A lack of beta globin and as consequently of hemoglobin results in an inability to supply adequate oxygen to the body, manifesting in beta thalassemia patients as 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 with a matched, most often a sibling donor. Patients with the more severe form of the disease, thalassemia major, require chronic blood transfusions to survive.
This content provides an overview of the beta thalassemia market, featuring a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. Current non-curative treatment options are limited to blood transfusion therapy which generates excessive total body iron and must be coupled with iron chelation therapeutics to prevent toxicity. Significant market opportunity remains for a universal curative treatment, as well as safe and tolerable treatments that can effectively manage symptoms such as anemia. The findings described in this content are derived from secondary research, best-in-class epidemiological analysis, and detailed interviews with experienced and European hematologists with expertise in diagnosing and treating beta thalassemia.
Beta thalassemia is a recessive monogenic blood disorder characterized by a lack of the protein beta globin; a key component of the oxygen transporting protein complex, hemoglobin. A lack of beta globin and hemoglobin results in an inability to supply adequate oxygen to the body, manifesting pathologically in beta thalassemia patients as anemia, failure to thrive, jaundice, pallor, poor musculature, and skeletal deformities. No curative therapy exists other than an allogenic hematopoietic stem cell transplantation with a matched sibling donor.
This report provides an overview of the beta thalassemia market, featuring a comprehensive analysis of patient populations, current therapies and medical practice, and opportunities for emerging therapies. Current non-curative treatment options are limited to blood transfusion therapy which generates excessive total body iron and must be coupled with iron chelation therapeutics to prevent toxicity. Hence, significant market opportunity remains for safe and tolerable treatments that can effectively manage symptoms, particularly in patients with the more severe form of the disease, thalassemia major. The findings described in this report are derived from secondary research, best-in-class epidemiological analysis, and detailed interviews with experienced and European hematologists with distinct expertise in diagnosing and treating beta thalassemia.