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 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), Global Blood Therapeutics’ Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) gives patients additional treatment options. Allogenic hematopoietic stem cell transplantation is the only available curative therapy, although Bluebird Bio’s emerging gene therapy lovo-cel and Vertex / CRISPR Therapeutics’ gene-edited cell therapy 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.
QUESTIONS ANSWERED
GEOGRAPHIES
United States and EU5
PRIMARY RESEARCH
EPIDEMIOLOGY
FORECAST
Drug-level sales and patient share of key sickle cell disease therapies through 2031.
EMERGING THERAPIES
Phase III / PR / approved: 8 drugs; Phase II and Phase I/II: > 10 drugs.
PRODUCT DESCRIPTION
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.
SOLUTION ENHANCEMENT
Niche & Rare Disease Landscape & Forecast introduces a new 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.