{"id":390237,"date":"2020-04-23T00:00:00","date_gmt":"2020-04-23T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfhe0004-2020-biopharma-sickle-cell-disease-niche-rare-disease-landscape-forecast-us-eu5-2020\/"},"modified":"2026-03-31T10:45:18","modified_gmt":"2026-03-31T10:45:18","slug":"nrlfhe0004-2020-biopharma-sickle-cell-disease-niche-rare-disease-landscape-forecast-us-eu5-2020","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfhe0004-2020-biopharma-sickle-cell-disease-niche-rare-disease-landscape-forecast-us-eu5-2020\/","title":{"rendered":"Sickle Cell Disease | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2020"},"content":{"rendered":"<p>Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin in red blood cells (RBCs) that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and vaso occlusive crisis (VOC) associated with pain. Most patients are managed with a combination of hydroxyurea, prophylactic penicillin, analgesics, and blood transfusions. The recent\u00a0FDA\u00a0approval of Global Blood Therapeutics\u2019 Oxbryta (voxelotor) and Novartis\u2019s Adakveo (crizanlizumab) and their expected commercial launches starting in 2020 will provide patients with additional disease management options. Allogenic\u00a0HSCT\u00a0with an\u00a0HLA-matched (most often sibling) donor is the only available curative therapy; however, pipeline gene therapies, such as Bluebird Bio\u2019s Zynteglo (LentiGlobin), are expected to offer additional, potentially curative options to patients. The most severe\u00a0HbSS\u00a0and\u00a0HbS\u03b20\u00a0patients suffer from painful episodes of\u00a0VOC, which substantially impacts quality of life. A high unmet exists for therapies that can reduce or eliminate\u00a0VOC\u00a0and extend life expectancy. Drug developers recognize the commercial opportunity in\u00a0SCD\u00a0and are focused on developing agents that target the\u00a0VOC\u00a0pain symptoms or the underlying genetic defect.<\/p>\n<p><strong>QUESTIONS ANSWERED<\/strong><\/p>\n<ul>\n<li>How large is the diagnosed prevalent\u00a0SCD\u00a0population in the United States and\u00a0EU5? How will the population change over the forecast period?<\/li>\n<li>What is the current treatment landscape, and how will it change in the next ten years? How will the launches of voxelotor, crizanlizumab, LentiGlobin, and docosahexaenoic acid impact\u00a0SCD\u00a0treatment?<\/li>\n<li>What pipeline molecules are promising? What sales \/ uptake could they secure in the\u00a0HbSS\u00a0and\u00a0HbS\u03b20\u00a0patient groups? How will new therapies impact medical practice?<\/li>\n<\/ul>\n<p><strong>PRODUCT DESCRIPTION<\/strong><\/p>\n<p><em>Niche &#038; Rare Disease Landscape &#038; Forecast:<\/em>\u00a0Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.<\/p>\n<p><strong>RELEASE DATE<\/strong><\/p>\n<p>April 2020<\/p>\n<p><strong>GEOGRAPHIES<\/strong><\/p>\n<p>United States and\u00a0EU5<\/p>\n<p><strong>PRIMARY RESEARCH:<\/strong><\/p>\n<ul>\n<li>Six country-specific interviews with thought-leading hematologists.<\/li>\n<li>Supported by survey data collected for this study.<\/li>\n<\/ul>\n<p><strong>EPIDEMIOLOGY:<\/strong><\/p>\n<p>Diagnosed prevalent and drug-treatable cases of sickle cell disease by country, segmented by clinical subtypes.<\/p>\n<p><strong>FORECAST<\/strong><\/p>\n<p>Drug-level sales and patient shares of key sickle cell disease therapies in 2029.<\/p>\n<p><strong>EMERGING THERAPIES<\/strong><\/p>\n<p>Phase III\/PR\/approved: 5 drugs; Phase II: 4 drugs. Coverage of select preclinical and Phase I products.<\/p>\n","protected":false},"template":"","class_list":["post-390237","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-hematology","biopharma-therapy-areas-sickle-cell-disease","biopharma-geography-us","biopharma-date-942"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390237","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\/390237\/revisions"}],"predecessor-version":[{"id":576528,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390237\/revisions\/576528"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390237"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}