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.
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PRODUCT DESCRIPTION
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.
RELEASE DATE
April 2020
GEOGRAPHIES
United States and EU5
PRIMARY RESEARCH:
EPIDEMIOLOGY:
Diagnosed prevalent and drug-treatable cases of sickle cell disease by country, segmented by clinical subtypes.
FORECAST
Drug-level sales and patient shares of key sickle cell disease therapies in 2029.
EMERGING THERAPIES
Phase III/PR/approved: 5 drugs; Phase II: 4 drugs. Coverage of select preclinical and Phase I products.