Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and vaso-occlusive crisis (VOC), which is associated with pain. Despite the launch of novel therapies, hydroxyurea is the mainstay first-line treatment of SCD in combination with prophylactic penicillin, analgesics, and blood transfusions. The FDA’s approval of Emmaus Life Sciences’ Endari (L-glutamine), Global Blood Therapeutics’ Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) has provided patients with additional disease management options. Allogenic hematopoietic stem cell transplantation has been the only cure, although Bluebird Bio’s emerging gene therapy, LentiGlobin, is expected to offer a possible cure for severe SCD. A high unmet need exists for therapies that can reduce or eliminate VOC in patients with the most severe HbSS and HbSβ0 and extend their life expectancy. Drug developers recognize this commercial opportunity and are focused on developing agents that target the VOC pain symptoms or the underlying genetic defect.
Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
United States and EU5
Diagnosed prevalent and drug-treatable cases of SCD by country, segmented by clinical subtype.
Drug-level sales and patient shares of key SCD therapies in 2030.
Phase III/PR/approved: 7 drugs; Phase II or I/II: >10 drugs.