{"id":390727,"date":"2018-03-16T00:00:00","date_gmt":"2018-03-16T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfon0008-2018-biopharma-myelodysplastic-syndromes-niche-rare-disease-landscape-forecast-us-eu5-2018\/"},"modified":"2026-04-21T05:27:22","modified_gmt":"2026-04-21T05:27:22","slug":"nrlfon0008-2018-biopharma-myelodysplastic-syndromes-niche-rare-disease-landscape-forecast-us-eu5-2018","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfon0008-2018-biopharma-myelodysplastic-syndromes-niche-rare-disease-landscape-forecast-us-eu5-2018\/","title":{"rendered":"Myelodysplastic Syndromes | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2018"},"content":{"rendered":"<p>Myelodysplastic syndromes ( MDS ) is a heterogeneous collection of hematopoietic stem cell disorders that result in cytopenias and risk of progression to acute myeloid leukemia ( AML ). Management of anemia through erythropoiesis-stimulating agents or more aggressive treatment with hypomethylating agents has long been the standard of care for lower- and higher-risk MDS patients, respectively. Development of therapies for patients who become refractory to standard-of-care treatments is the key unmet need for this market. The MDS pipeline is robust, and several projected market entrants are poised to penetrate subpopulations of the lower-risk MDS market. While development of novel agents for higher-risk MDS patients is active, this segment of the market has proved to be challenging for developers to demonstrate overall survival benefit.\u00a0<br \/>\nQuestions Answered:\u00a0<\/p>\n<ul>\n<li>How do interviewed experts perceive currently available MDS therapies?\u00a0<\/li>\n<li>Will further research into genetic markers impact either diagnosis or prognosis in MDS ?\u00a0<\/li>\n<li>What emerging therapies are most likely to impact the MDS market in 2026?\u00a0\u00a0<\/li>\n<li>How will Celgene\u2019s oral azacitidine ( CC -486) be received by physicians? What will be the off-label use if the label is restricted to lower-risk patients?\u00a0<\/li>\n<li>Will Celgene\u2019s luspatercept ( ACE -536) be limited to a second-line option for lower-risk MDS patients?\u00a0<\/li>\n<li>What therapies currently marketed or are in late-phase development for AML will have relevance in the MDS market?\u00a0<\/li>\n<\/ul>\n<p>\n<strong>Scope\u00a0<\/strong><\/p>\n<p>Markets covered : United States, France, Germany, Italy, Spain, and the United Kingdom.\u00a0<br \/>\nPrimary research : Six country-specific interviews with thought leading hematologists.\u00a0<br \/>\nEpidemiology : Incident and diagnosed prevalent cases of MDS by country, morphology, and prognostic risk; including drug-treated cases.\u00a0<br \/>\nMarket forecast : Drug-level sales and patient share of key MDS therapies in 2026.\u00a0<br \/>\nEmerging therapies : Phase III: 7 drugs. Phase II: 21 drugs. Coverage of select Phase I\/II and I products.\u00a0<br \/>\nKey companies : Celgene, Otsuka, Amgen, Janssen,\u00a0Acceleron, Novartis, Agios, Jazz, Celator, Onconova, Astex.\u00a0<br \/>\nKey drugs : Azacitidine (Vidaza, generics), decitabine (Dacogen, generics),\u00a0Revlimid (lenalidomide), epoetin alfa (Epogen, Procrit), darbepoetin alfa (Aranesp), CC -486, luspatercept, eltrombopag (Promacta\/Revolade), enasidenib, CPX-351 (Vyxeos), rigosertib (Estybon), guadecitabine.\u00a0<\/p>\n<h3>Questions Answered in This Report:<\/h3>\n<ul>\n<li>\n<p>How do interviewed experts perceive currently available MDS therapies?\u00a0<br \/>\n\tWill further research into genetic markers impact either diagnosis or prognosis in MDS ?\u00a0<br \/>\n\tWhat emerging therapies are most likely to impact the MDS market in 2026?\u00a0\u00a0<br \/>\n\tHow will Celgene\u2019s oral azacitidine ( CC -486) be received by physicians? What will be the off-label use if the label is restricted to lower-risk patients?\u00a0<br \/>\n\tWill Celgene\u2019s luspatercept ( ACE -536) be limited to a second-line option for lower-risk MDS patients?\u00a0<br \/>\n\tWhat therapies currently marketed or are in late-phase development for AML will have relevance in the MDS market?\u00a0<\/p>\n<\/li>\n<\/ul>\n<h3>Scope:<\/h3>\n<p>Myelodysplastic syndromes ( MDS ) is a heterogeneous collection of hematopoietic stem cell disorders that result in cytopenias and risk of progression to acute myeloid leukemia ( AML ). Management of anemia through erythropoiesis-stimulating agents or more aggressive treatment with hypomethylating agents has long been the standard of care for lower- and higher-risk MDS patients, respectively. Development of therapies for patients who become refractory to standard-of-care treatments is the key unmet need for this market. The MDS pipeline is robust, and several projected market entrants are poised to penetrate subpopulations of the lower-risk MDS market. While development of novel agents for higher-risk MDS patients is active, this segment of the market has proved to be challenging for developers to demonstrate overall survival benefit.<\/p>\n<p>\n<strong>Markets covered :<\/strong>\u00a0United States, France, Germany, Italy, Spain, and the United Kingdom.\u00a0<br \/>\nPrimary research : Six country-specific interviews with thought leading hematologists.\u00a0<br \/>\nEpidemiology : Incident and diagnosed prevalent cases of MDS by country, morphology, and prognostic risk; including drug-treated cases.\u00a0<br \/>\nMarket forecast : Drug-level sales and patient share of key MDS therapies in 2026.\u00a0<br \/>\nEmerging therapies : Phase III: 7 drugs. Phase II: 21 drugs. Coverage of select Phase I\/II and I products.\u00a0<br \/>\nKey companies : Celgene, Otsuka, Amgen, Janssen,\u00a0Acceleron, Novartis, Agios, Jazz, Celator, Onconova, Astex.\u00a0<br \/>\nKey drugs : Azacitidine (Vidaza, generics), decitabine (Dacogen, generics),\u00a0Revlimid (lenalidomide), epoetin alfa (Epogen, Procrit), darbepoetin alfa (Aranesp), CC -486, luspatercept, eltrombopag (Promacta\/Revolade), enasidenib, CPX-351 (Vyxeos), rigosertib (Estybon), guadecitabine.\u00a0<\/p>\n","protected":false},"template":"","class_list":["post-390727","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-myelodysplastic-syndromes","biopharma-geography-us","biopharma-date-920"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390727","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\/390727\/revisions"}],"predecessor-version":[{"id":576750,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390727\/revisions\/576750"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390727"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}