{"id":390963,"date":"2017-04-28T00:00:00","date_gmt":"2017-04-28T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfmd0004-2017-biopharma-primary-biliary-cholangitis-niche-rare-disease-landscape-forecast-us-eu5-2017\/"},"modified":"2026-04-27T23:42:48","modified_gmt":"2026-04-27T23:42:48","slug":"nrlfmd0004-2017-biopharma-primary-biliary-cholangitis-niche-rare-disease-landscape-forecast-us-eu5-2017","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfmd0004-2017-biopharma-primary-biliary-cholangitis-niche-rare-disease-landscape-forecast-us-eu5-2017\/","title":{"rendered":"Primary Biliary Cholangitis | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2017"},"content":{"rendered":"<p>As the first agent to be approved for primary biliary cholangitis (<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>), ursodeoxycholic acid (<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>) was launched nearly two decades ago and transformed the treatment of this disease by significantly reducing mortality rates. However, there remains a notable subset of\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0patients who are currently underserved by\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>. Now, given the medical advances seen for treatment of other liver disorders, there is renewed interest in the development of new\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0agents to address areas of unmet need in this market. Intercept Pharmaceuticals hopes to capitalize on the need for new\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0agents, specifically for patients who do not respond to\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>. Intercept\u2019s novel\u00a0<abbr title=\"farnesoid X receptor\">FXR<\/abbr>\u00a0agonist obeticholic acid (Ocaliva) was approved in the United States and Europe in 2016 and is expected to gain market share primarily among\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>\u00a0nonresponders. However, pipeline agents have the potential to improve upon both\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>\u00a0and Ocaliva, and thus could gain share in what will be a highly contested market.<\/p>\n<p><strong>Questions Answered:<\/strong><\/p>\n<ul>\n<li>For nearly 20 years, there was only therapy approved for PBC.\u00a0<strong>How effective is the mainstay of therapy,\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>, in treating\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0patients? Does any unmet need remain for patients who respond to\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>?\u00b7<\/strong><\/li>\n<li>In 2016, Intercept Pharmaceuticals\u2019 Ocaliva became the first-ever\u00a0<abbr title=\"farnesoid X receptor\">FXR<\/abbr>-agonist approved for any indication.\u00a0<strong>How will uptake of Intercept Pharmaceuticals\u2019 Ocaliva impact management of\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0patients in the United States and the\u00a0<abbr title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>?\u00a0<\/strong><\/li>\n<li>There are now two agents approved for\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>, and when they are used in combination, they may radically alter clinical outcomes in patients who previously did not show benefit.\u00a0<strong>With the availability of\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>\u00a0and Ocaliva, what areas of unmet need remain for the treatment of\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>? What improvements can emerging agents offer in the management of\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>?<\/strong><\/li>\n<li><abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0is a chronic, progressive disease where the most important clinical end points take many years to reach.\u00a0<strong>What clinical end points do emerging agents need to meet in order to compete effectively against\u00a0<abbr title=\"ursodeoxycholic acid\">UDCA<\/abbr>\u00a0and Ocaliva?<\/strong><\/li>\n<\/ul>\n<p><strong>Markets covered:<\/strong>\u00a0United States, France, Germany, Italy, Spain, and United Kingdom.<\/p>\n<p><strong>Primary research:<\/strong>\u00a0Six country-specific interviews with\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0experts. Surveys were conducted in the United States and Europe to determine patient share estimates and treatment rates.<\/p>\n<p><strong>Epidemiology:<\/strong>\u00a0Diagnosed prevalent cases, incident cases; diagnosed drug-treated cases. Emerging therapies: Phase II: 8 drugs.<\/p>\n<p><strong>Market forecast features:<\/strong>\u00a0Single-point patient-based market forecast extending for 2026.<\/p>\n<p><strong>Key companies:<\/strong>\u00a0Intercept Pharmaceuticals, Gilead, NGM Biopharmaceuticals, Novartis, Shire, GSK, CymaBay, and Gilead.<\/p>\n<p><strong>Key drugs:<\/strong>\u00a0Obeticholic acid (Ocaliva), NGM-282, seladelpar, lopixibat, GSK-2330672, GS-9674.<\/p>\n<p><strong>Recent events:<\/strong>\u00a0The recent launch of Ocaliva, an innovative new\u00a0<abbr title=\"farnesoid X receptor\">FXR<\/abbr>-agonist, has dramatic implications for the market and related agents in earlier-stage development. In this report, we analyze physician perception of Ocaliva, its likely positioning and uptake, and its implications on emerging\u00a0<abbr title=\"primary\u00a0biliary\u00a0cirrhosis\/cholangitis\">PBC<\/abbr>\u00a0therapeutics.<\/p>\n","protected":false},"template":"","class_list":["post-390963","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-primary-biliary-cholangitis","biopharma-geography-us","biopharma-date-890"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390963","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\/390963\/revisions"}],"predecessor-version":[{"id":576835,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390963\/revisions\/576835"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390963"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}