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.
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