Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin (Hb) in red blood cells that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and painful vaso-occlusive crisis (VOC). Despite the launch of novel therapies, hydroxyurea is the mainstay first-line treatment for SCD, in combination with prophylactic penicillin, analgesics, and blood transfusions. The FDA’s approval of Emmaus Life Sciences’ Endari (L-glutamine), Pfizer’s (formerly Global Blood Therapeutics’ [GBT’s]) Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) gives patients additional treatment options. Allogenic hematopoietic stem cell transplantation is the only available curative therapy, although gene therapies such as Bluebird Bio’s lovo-cel and CRISPR / Vertex’s exa-cel have the potential to cure severe SCD. Therapies that can reduce or eliminate VOCs in the most severely ill HbSS and HbSβ0 patients and extend life expectancy are strongly needed. Recognizing the commercial opportunity, drug developers are focused on developing agents that target VOC pain and/or the underlying genetic defect.
Geographies: United States and EU5.
Primary research: Six country-specific interviews with thought-leading hematologists. Supported by survey data collected for this and other Clarivate research.
Epidemiology: Diagnosed prevalent and drug-treatable cases of sickle cell disease by country. Diagnosed prevalent cases of sickle cell disease segmented by clinical subtype.
Forecast: Drug-level sales and patient share of key sickle cell disease therapies in 2032.
Emerging therapies: Phase 3 / PR / approved: 9 drugs; Phase 2 and Phase 1/2: > 10 drugs.
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.
Niche & Rare Disease Landscape & Forecast introduces a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.