Sickle Cell Disease | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2022

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

• 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 10 years? How will the launches of recently approved therapies (voxelotor and crizanlizumab) and late-phase therapies (lovo-cel, exa-cel, Forma Therapeutics’ etavopivat, Global Blood Therapeutics’ inclacumab, and Agios’s mitapivat) affect SCD treatment?
• What sales / uptake could emerging therapies secure in the HbSS and HbSβ0 patient groups? How will new therapies be incorporated into medical practice?

GEOGRAPHIES

United States and EU5

PRIMARY RESEARCH

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

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 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.