{"id":517304,"date":"2025-08-18T00:00:00","date_gmt":"2025-08-18T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfhe0004-2025-biopharma-sickle-cell-disease-landscape-forecast-niche-rare-disease-landscape-forecast-us-eu5\/"},"modified":"2026-03-31T10:25:21","modified_gmt":"2026-03-31T10:25:21","slug":"nrlfhe0004-2025-biopharma-sickle-cell-disease-landscape-forecast-niche-rare-disease-landscape-forecast-us-eu5","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfhe0004-2025-biopharma-sickle-cell-disease-landscape-forecast-niche-rare-disease-landscape-forecast-us-eu5\/","title":{"rendered":"Sickle Cell Disease &#8211; Landscape &#038; Forecast &#8211; Niche &#038; Rare Disease Landscape &#038; Forecast (US\/EU5)"},"content":{"rendered":"<p>Sickle cell disease (<abbr title=\"sickle cell disease\">SCD<\/abbr>) 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 (<abbr title=\"vaso-occlusive crisis\">VOC<\/abbr>s). Hydroxyurea is the mainstay first-line treatment for <abbr title=\"sickle cell disease\">SCD<\/abbr>, in combination with prophylactic penicillin, analgesics, and blood transfusions. The <abbr title=\"Food and Drug Administration\">FDA<\/abbr>\u2019s approval of Emmaus Life Sciences\u2019 L-glutamine (Endari), Pfizer\u2019s voxelotor (Oxbryta), and Novartis\u2019s crizanlizumab (Adakveo) gave patients additional treatment options. However, the subsequent withdrawal of approved therapies has left patients with limited options. For instance, Pfizer\u2019s voxelotor was withdrawn from global markets in 2024, and the conditional marketing authorization of Novartis\u2019s crizanlizumab was revoked by the <abbr title=\"European Commission\">EC<\/abbr> 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\u2019s exagamglogene autotemcel (Casgevy), approved by the <abbr title=\"Food and Drug Administration\">FDA<\/abbr> and <abbr title=\"European Commission\">EC<\/abbr> (conditionally approved), and Bluebird Bio\u2019s <abbr title=\"Food and Drug Administration\">FDA<\/abbr>-approved lovotibeglogene autotemcel (Lyfgenia) are gene therapies that may be able to cure severe <abbr title=\"sickle cell disease\">SCD<\/abbr>. Therapies that can reduce or eliminate <abbr title=\"vaso-occlusive crisis\">VOC<\/abbr>s in the most severely ill patients and extend life expectancy are still strongly needed.<\/p>\n<p><strong>Questions answered<\/strong><\/p>\n<ul class=\"round-bullets\">\n<li>How large is the diagnosed prevalent <abbr title=\"sickle cell disease\">SCD<\/abbr> population in the United States and <abbr title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>, and how will it change over the forecast period?<\/li>\n<li>What is the current treatment landscape, and how will it change in the next 10 years? How will the launches of gene therapies affect <abbr title=\"sickle cell disease\">SCD<\/abbr> treatment?<\/li>\n<li>What sales \/ uptake could emerging therapies secure in the <abbr title=\"homozygous sickle hemoglobin\">HbSS<\/abbr> and <abbr title=\"hemoglobin S\">HbS<\/abbr>\u03b20 patient groups?<\/li>\n<\/ul>\n<p><strong>Geographies: <\/strong>United States and <abbr title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>.<\/p>\n<p><strong>Primary research: <\/strong>Country-specific interviews with thought-leading hematologists. Supported by survey data collected for this study<\/p>\n<p><strong>Epidemiology: <\/strong>Diagnosed prevalent and drug-treatable cases of <abbr data-abbreviation-entity=\"6230\" title=\"sickle cell disease\">SCD<\/abbr> by country; diagnosed prevalent cases of <abbr data-abbreviation-entity=\"6230\" title=\"sickle cell disease\">SCD<\/abbr> by country, segmented by clinical subtype<\/p>\n<p><strong>Forecast: <\/strong>Drug-level sales and patient share of key <abbr data-abbreviation-entity=\"6230\" title=\"sickle cell disease\">SCD<\/abbr> therapies in 2024 and 2034<\/p>\n<p><strong>Drug treatments: <\/strong>Coverage of key current and emerging therapies<\/p>\n<p><strong>Product description: <\/strong><em>Niche &#038; Rare Disease Landscape &#038; Forecast <\/em>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:<\/p>\n<ul>\n<li>Optimize your long-term disease and development strategy.<\/li>\n<li>Quantify market potential for your pipeline assets and those of your competitors.<\/li>\n<li>Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.<\/li>\n<li>Gauge the commercial outlook and impact of key market events.<\/li>\n<\/ul>\n","protected":false},"template":"","class_list":["post-517304","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-hematology","biopharma-therapy-areas-sickle-cell-disease","biopharma-geography-us","biopharma-date-2297"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/517304","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report"}],"about":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/types\/report"}],"version-history":[{"count":2,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/517304\/revisions"}],"predecessor-version":[{"id":575742,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/517304\/revisions\/575742"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=517304"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}