Sickle Cell Disease | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2020

Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells (RBCs) that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and vaso occlusive crisis (VOC) associated with pain. Most patients are managed with a combination of hydroxyurea, prophylactic penicillin, analgesics, and blood transfusions. The recent FDA approval of Global Blood Therapeutics’ Oxbryta (voxelotor) and Novartis’s Adakveo (crizanlizumab) and their expected commercial launches starting in 2020 will provide patients with additional disease management options. Allogenic HSCT with an HLA-matched (most often sibling) donor is the only available curative therapy; however, pipeline gene therapies, such as Bluebird Bio’s Zynteglo (LentiGlobin), are expected to offer additional, potentially curative options to patients. The most severe HbSS and HbSβ0 patients suffer from painful episodes of VOC, which substantially impacts quality of life. A high unmet exists for therapies that can reduce or eliminate VOC and extend life expectancy. Drug developers recognize the commercial opportunity in SCD and are focused on developing agents that target the VOC pain symptoms or the underlying genetic defect.


  • How large is the diagnosed prevalent SCD population in the United States and EU5? How will the population change over the forecast period?
  • What is the current treatment landscape, and how will it change in the next ten years? How will the launches of voxelotor, crizanlizumab, LentiGlobin, and docosahexaenoic acid impact SCD treatment?
  • What pipeline molecules are promising? What sales / uptake could they secure in the HbSS and HbSβ0 patient groups? How will new therapies impact 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.


April 2020


United States and EU5


  • Six country-specific interviews with thought-leading hematologists.
  • Supported by survey data collected for this study.


Diagnosed prevalent and drug-treatable cases of sickle cell disease by country, segmented by clinical subtypes.


Drug-level sales and patient shares of key sickle cell disease therapies in 2029.


Phase III/PR/approved: 5 drugs; Phase II: 4 drugs. Coverage of select preclinical and Phase I products.

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