{"id":390897,"date":"2017-09-29T00:00:00","date_gmt":"2017-09-29T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfim0001-2017-biopharma-cystic-fibrosis-niche-rare-disease-landscape-forecast-us-eu5-2017\/"},"modified":"2026-03-31T10:49:49","modified_gmt":"2026-03-31T10:49:49","slug":"nrlfim0001-2017-biopharma-cystic-fibrosis-niche-rare-disease-landscape-forecast-us-eu5-2017","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfim0001-2017-biopharma-cystic-fibrosis-niche-rare-disease-landscape-forecast-us-eu5-2017\/","title":{"rendered":"Cystic Fibrosis | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2017"},"content":{"rendered":"<p>Cystic fibrosis (<abbr data-original-title=\"cystic fibrosis\" title=\"\">CF<\/abbr>) is a genetic disease caused by any one of the more than 2,000 mutations identified in the cystic fibrosis transmembrane conductance regulator (<em><abbr data-original-title=\"cystic fibrosis transmembrane conductance regulator\" title=\"\">CFTR<\/abbr><\/em>) gene.\u00a0<em><abbr data-original-title=\"cystic fibrosis transmembrane conductance regulator\" title=\"\">CFTR<\/abbr><\/em>\u00a0mutations lead to aberrant chloride transport in a variety of tissues, most notably the lungs and pancreas. As a result,\u00a0<abbr data-original-title=\"cystic fibrosis\" title=\"\">CF<\/abbr>\u00a0patients generally suffer from pancreatic damage, which affects metabolism and nutrient absorption. Chronic respiratory problems, such as persistent lung infection stemming from the accumulation of thick, viscous mucus in the lungs, can result in respiratory failure\u2014the leading cause of death in\u00a0<abbr data-original-title=\"cystic fibrosis\" title=\"\">CF<\/abbr>. Historically, therapeutic options for\u00a0<abbr data-original-title=\"cystic fibrosis\" title=\"\">CF<\/abbr>\u00a0were limited to therapies to dislodge mucus buildup, inhaled antibiotics, and pancreatic replacement therapy. With the market entry of Vertex Pharmaceuticals\u2019 Kalydeco, a small-molecule\u00a0<abbr data-original-title=\"cystic fibrosis transmembrane conductance regulator\" title=\"\">CFTR<\/abbr>\u00a0potentiator, and Orkambi, a combination of Kalydeco and the\u00a0<abbr data-original-title=\"cystic fibrosis transmembrane conductance regulator\" title=\"\">CFTR<\/abbr>\u00a0corrector lumacaftor, disease-modifying therapy (<abbr data-original-title=\"disease-modifying therapy\" title=\"\">DMT<\/abbr>) has become a reality for a subset of\u00a0<abbr data-original-title=\"cystic fibrosis\" title=\"\">CF<\/abbr>\u00a0patients. Pipeline agents, including next-generation combinations, seek to improve on the performance of marketed\u00a0<abbr data-original-title=\"disease-modifying therapy\" title=\"\">DMT<\/abbr>s (e.g., Vertex\u2019s ivacaftor\/tezacaftor) or further expand the reach of\u00a0<abbr data-original-title=\"disease-modifying therapy\" title=\"\">DMT<\/abbr>\u00a0in the\u00a0<abbr data-original-title=\"cystic fibrosis\" title=\"\">CF<\/abbr>\u00a0population (e.g., triple combinations).<\/p>\n","protected":false},"template":"","class_list":["post-390897","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-cystic-fibrosis","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\/390897","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\/390897\/revisions"}],"predecessor-version":[{"id":576814,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390897\/revisions\/576814"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390897"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}