Sickle Cell Disease – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

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’ L-glutamine (Endari), Pfizer’s voxelotor (Oxbryta), and Novartis’s crizanlizumab (Adakveo) gave patients additional treatment options. However, the subsequent withdrawal of approved therapies has left patients with limited options. For instance, Pfizer’s voxelotor was withdrawn from global markets in 2024, and the conditional marketing authorization of Novartis’s crizanlizumab was revoked by the EC in 2023; both of these agents initially provided patients with meaningful, targeted treatment choices. Allogenic hematopoietic stem cell transplantation is the only curative therapy but is limited owing to the lack of donors. CRISPR / Vertex’s exagamglogene autotemcel (Casgevy), approved by the FDA and EC (conditionally approved), and Bluebird Bio’s FDA-approved lovotibeglogene autotemcel (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 gene therapies affect SCD treatment?
• What sales / uptake could emerging therapies secure in the HbSS and HbSβ0 patient groups?

Geographies: United States and EU5.

Primary research: 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 in 2024 and 2034

Drug treatments: Coverage of key current and emerging therapies

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

• Optimize your long-term disease and development strategy.
• Quantify market potential for your pipeline assets and those of your competitors.
• Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.
• Gauge the commercial outlook and impact of key market events.