Sickle Cell Disease | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2024

Sickle cell disease (SCD) is a rare genetic blood disorder characterized by the 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 crises (VOCs). 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, Pfizer’s Oxbryta, and Novartis’s Adakveo gives patients additional treatment options, although subsequent withdrawals have curtailed this trend. Allogenic hematopoietic stem cell transplantation is the only curative therapy but is limited due to the lack of donors. CRISPR / Vertex’s Casgevy, approved by the FDA and EC (conditionally approved), and Bluebird Bio’s FDA-approved Lyfgenia are gene therapies that may be able to cure severe SCD. Therapies that can reduce or eliminate VOCs in the most severely ill patients and extend life expectancy are still strongly needed.

Questions answered

• How large is the diagnosed prevalent SCD population in the United States and EU5, and how will it 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 gene therapies (Casgevy and Lyfgenia) affect SCD treatment?
• What sales / uptake could emerging therapies secure in the HbSS and HbSβ0 patient groups?

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 SCD by country, diagnosed prevalent cases of SCD by country, segmented by clinical subtype.

Forecast: Drug-level sales and patient share of key SCD therapies through 2033.

Drug treatments: Coverage of key current and emerging therapies.

Product description: Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research.

Key feature: Niche & Rare Disease Landscape & Forecast features 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.