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

Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin (Hb) 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), Pfizer’s (formerly Global Blood Therapeutics’ [GBT’s]) Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) gives patients additional treatment options. Allogenic hematopoietic stem cell transplantation is the only available curative therapy, although gene therapies such as Bluebird Bio’s lovo-cel and CRISPR / Vertex’s 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, crizanlizumab, lovo-cel, and exa-cel) and late-phase therapies (Novo Nordisk’s [formerly Forma Therapeutics’] etavopivat, Pfizer’s [formerly GBT’s] inclacumab, Agios’s Pyrukynd [mitapivat], and Pfizer’s PF-07940367) impact 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 and other Clarivate research.

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 in 2032.

Emerging therapies: Phase 3 / PR / approved: 9 drugs; Phase 2 and Phase 1/2: > 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 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.