{"id":389784,"date":"2022-09-15T00:00:00","date_gmt":"2022-09-15T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfhe0004-2022-biopharma-sickle-cell-disease-niche-rare-disease-landscape-forecast-us-eu5-2022\/"},"modified":"2026-03-31T10:38:31","modified_gmt":"2026-03-31T10:38:31","slug":"nrlfhe0004-2022-biopharma-sickle-cell-disease-niche-rare-disease-landscape-forecast-us-eu5-2022","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfhe0004-2022-biopharma-sickle-cell-disease-niche-rare-disease-landscape-forecast-us-eu5-2022\/","title":{"rendered":"Sickle Cell Disease | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2022"},"content":{"rendered":"<p>Sickle cell disease (SCD) is a rare genetic blood disorder characterized by 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 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\u2019s approval of Emmaus Life Sciences\u2019 Endari (L-glutamine), Global Blood Therapeutics\u2019 Oxbryta (voxelotor), and Novartis\u2019s Adakveo (crizanlizumab) gives patients additional treatment options. Allogenic hematopoietic stem cell transplantation is the only available curative therapy, although Bluebird Bio\u2019s emerging gene therapy lovo-cel and Vertex \/ CRISPR Therapeutics\u2019 gene-edited cell therapy exa-cel have the potential to cure severe SCD. Therapies that can reduce or eliminate VOCs in the most severely ill HbSS and HbS\u03b20 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.<\/p>\n<p><strong>QUESTIONS ANSWERED<\/strong><\/p>\n<ul>\n<li>How large is the diagnosed prevalent SCD population in the United States and EU5? 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 10 years? How will the launches of recently approved therapies (voxelotor and crizanlizumab) and late-phase therapies (lovo-cel, exa-cel, Forma Therapeutics\u2019 etavopivat, Global Blood Therapeutics\u2019 inclacumab, and Agios\u2019s mitapivat) affect SCD treatment?<\/li>\n<li>What sales \/ uptake could emerging therapies secure in the HbSS and HbS\u03b20 patient groups? How will new therapies be incorporated into medical practice?<\/li>\n<\/ul>\n<p><strong>GEOGRAPHIES<\/strong><\/p>\n<p>United States and EU5<\/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<ul>\n<li>Diagnosed prevalent and drug-treatable cases of sickle cell disease by country<\/li>\n<li>Diagnosed prevalent cases of sickle cell disease segmented by clinical subtype.<\/li>\n<\/ul>\n<p><strong>FORECAST <\/strong><\/p>\n<p>Drug-level sales and patient share of key sickle cell disease therapies through 2031.<\/p>\n<p><strong>EMERGING THERAPIES<\/strong><\/p>\n<p>Phase III \/ PR \/ approved: 8 drugs; Phase II and Phase I\/II: > 10 drugs.<\/p>\n<p><strong>PRODUCT DESCRIPTION<\/strong><\/p>\n<p>Niche &#038; Rare Disease Landscape &#038; 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.<\/p>\n<p><strong>SOLUTION ENHANCEMENT<\/strong><\/p>\n<p>Niche &#038; Rare Disease Landscape &#038; Forecast introduces a new 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.<\/p>\n","protected":false},"template":"","class_list":["post-389784","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-hematology","biopharma-therapy-areas-sickle-cell-disease","biopharma-geography-us","biopharma-date-960"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/389784","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\/389784\/revisions"}],"predecessor-version":[{"id":576209,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/389784\/revisions\/576209"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=389784"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}