{"id":390616,"date":"2018-08-02T00:00:00","date_gmt":"2018-08-02T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfmd0002-2018-biopharma-amyloidosis-niche-rare-disease-landscape-forecast-us-eu5-2018\/"},"modified":"2026-04-27T11:27:35","modified_gmt":"2026-04-27T11:27:35","slug":"nrlfmd0002-2018-biopharma-amyloidosis-niche-rare-disease-landscape-forecast-us-eu5-2018","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfmd0002-2018-biopharma-amyloidosis-niche-rare-disease-landscape-forecast-us-eu5-2018\/","title":{"rendered":"Amyloidosis | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2018"},"content":{"rendered":"<p>Amyloidosis\u00a0is caused by the deposition of insoluble amyloid fibrils formed by the accumulation of misfolded proteins into affected organs. Amyloid immunoglobulin light chain (AL), amyloid A (AA), and amyloid transthyretin (ATTR) amyloidosis are the three major subtypes of the disease. The treatment of\u00a0AL\u00a0amyloidosis is derived from the chemotherapy regimens used to treat multiple myeloma, while the treatment of\u00a0AA\u00a0amyloidosis requires the management of the underlying inflammation that leads to the disease, through off-label use of\u00a0IL-6\/IL-1 inhibitors.\u00a0ATTR\u00a0amyloidosis is managed using\u00a0TTR\u00a0tetramer stabilizers tafamidis (Pfizer\u2019s Vyndaqel) and diflunisal (generics), the doxycycline\/TUDCA\u00a0(generics) combination, and symptomatic treatment of cardiomyopathy. Tafamidis, approved in Europe for polyneuropathy caused by hereditary\u00a0ATTR\u00a0amyloidosis(ATTR\u2013FAP), is the only approved drug for any amyloidosis subtype. Although there is a dearth of approved drugs for amyloidosis, several pharmacotherapies are in late-stage development for the disease.\u00a0RNA-inhibiting therapies\u2014Akcea Therapeutics and Ionis Pharmaceuticals\u2019 inotersen and Alnylam\u2019s Pharmaceuticals patisiran\u2014are preregistered for hereditary\u00a0ATTR\u00a0amyloidosis in the United States; and while patisiran is also preregistered in Europe, inotersen has been approved in the European Union for\u00a0ATTR\u2013FAP. Tafamidis, approved in Europe for\u00a0ATTR\u2013FAP, is in Phase III development for\u00a0ATTR-associated cardiomyopathy. Moreover, Takeda Oncology\u2019s oral proteasome inhibitor, ixazomib (Ninlaro), and Janssen Biotech and Genmab\u2019s daratumumab (Darzalex), an anti-CD38 monoclonal antibody, are in late-phase development for\u00a0AL\u00a0amyloidosis. Despite available treatment options and various therapies in late-phase development, there will remain a high unmet need for additional and effective therapies for amyloidosis through 2027.<\/p>\n<p><strong>Questions Answered<\/strong><\/p>\n<ul>\n<li>How will the sizes of the\u00a0AL,\u00a0AA\u00a0and\u00a0ATTR\u00a0amyloidosis populations across the United States and the\u00a0EU5\u00a0change through 2027?<\/li>\n<li>What are key current therapies in the amyloidosis market?<\/li>\n<li>What are the key drug targets emerging from basic and clinical research in amyloidosis? Which emerging therapies do amyloidosis experts consider most promising? How would new therapies influence the management of amyloidosis patients?<\/li>\n<li>How are emerging amyloidosis therapies being evaluated by the amyloidosis experts across the United States and the\u00a0EU5, and which are likely to launch by 2027? What commercial impact will they have on the amyloidosis market?<\/li>\n<\/ul>\n<p><strong>Product\u00a0<\/strong><strong>Description<\/strong><\/p>\n<p>Niche &#038; Rare Disease Landscape &#038; Forecast: Comprehensive 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","protected":false},"template":"","class_list":["post-390616","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-amyloidosis","biopharma-therapy-areas-cardiovascular","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\/390616","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\/390616\/revisions"}],"predecessor-version":[{"id":576719,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390616\/revisions\/576719"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390616"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}