{"id":390967,"date":"2017-03-30T00:00:00","date_gmt":"2017-03-30T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0005-2017-biopharma-amyotrophic-lateral-sclerosis-niche-rare-disease-landscape-forecast-us-eu5-2017\/"},"modified":"2026-04-17T11:41:06","modified_gmt":"2026-04-17T11:41:06","slug":"nrlfcg0005-2017-biopharma-amyotrophic-lateral-sclerosis-niche-rare-disease-landscape-forecast-us-eu5-2017","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0005-2017-biopharma-amyotrophic-lateral-sclerosis-niche-rare-disease-landscape-forecast-us-eu5-2017\/","title":{"rendered":"Amyotrophic Lateral Sclerosis | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2017"},"content":{"rendered":"<p>Amyotrophic lateral sclerosis (<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>) is a degenerative disorder of motor neurons that leads to progressive muscle wasting and weakness, with a typical survival time of three to five years from onset. Riluzole (Concordia Health\/Sanofi\u2019s Rilutek, generics) is the only approved disease-modifying therapy (<abbr title=\"disease-modifying therapy\">DMT<\/abbr>) for\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>, although interviewed experts consider the drug\u2019s efficacy to be modest. Experts emphasize the unmet need for additional, more effective\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0<abbr title=\"disease-modifying therapy\">DMT<\/abbr>s, as well as new symptomatic drugs, and biomarkers to aid in diagnosis, prognosis, and pharmacodynamics. The\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0clinical pipeline is robust and diverse, comprising late-stage programs aimed at enhancing muscle function, immune modulation, and neuroprotection, as well as mid\u2013 or early-stage gene therapy or gene silencing approaches, stem-cell therapies, and immune-targeting agents\u2014and experts contend that regimens combining multiple mechanisms of action could hold significant clinical promise in the management of\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>. Thus, considerable commercial opportunity exists for developers that can overcome the scientific and clinical hurdles blocking the path to regulatory approval for the treatment of this devastating disease.<\/p>\n<p><strong>Questions Answered:<\/strong><\/p>\n<ul>\n<li>Amyotrophic Lateral Sclerosis (<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>) is a rare disease affecting only a small percentage of the population.\u00a0<strong>How large is diagnosed prevalent\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0population in the United States and\u00a0<abbr title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>\u00a0(France, Germany, Italy, Spain, and the United Kingdom)? What percentage of the\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0population has a familial versus sporadic form of the disease? What percentage is afflicted with comorbid frontotemporal dementia? How will the size of the\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0population change over the 2016-2026 forecast period? What percentage of diagnosed\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0patients receives drug treatment?<\/strong><\/li>\n<li>With only a single drug approved to prolong survival and maintain function, a high level of unmet need for new\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0treatments exists.\u00a0<strong>What are the key drug targets emerging from basic and clinical research in\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>? What are the most pressing gaps in treatment identified by interviewed\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0experts? How well will emerging therapies in the\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0pipeline address key unmet treatment needs?<\/strong><\/li>\n<li>The\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0pipeline comprises therapies with diverse mechanisms of action and technologies.\u00a0<strong>How are emerging therapies evaluated in clinical trials, and which programs do interviewed experts consider the most promising? Which emerging therapies are likely to launch by 2026, and what clinical and commercial impact will they have?<\/strong><\/li>\n<\/ul>\n<p><strong>Scope:<\/strong><\/p>\n<ul>\n<li><strong>Market covered:\u00a0<\/strong>United States, France, Germany, Italy, Spain, and the United Kingdom.<\/li>\n<li><strong>Primary research:<\/strong>\u00a0Six country-specific interviews with\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>\u00a0neurologists.<\/li>\n<li><strong>Epidemiology:<\/strong>\u00a0Diagnosed prevalence, diagnosed prevalence by disease type (familial or sporadic\u00a0<abbr title=\"amyotrophic lateral sclerosis\">ALS<\/abbr>), diagnosed prevalence by comorbid frontotemporal dementia.<\/li>\n<li><strong>Emerging therapies:<\/strong>\u00a0Phase III\/<abbr title=\"preregistered\">PR<\/abbr>: 3; Phase II: 8; Phase I\/II: 2; coverage of select preclinical and Phase I products.<\/li>\n<li><b>Market forecast and alternative market scenarios:\u00a0<\/b>Drug-level sales and patient share of ALS therapies in 2026.<\/li>\n<li><b>Key companies:\u00a0<\/b>Cytokinetics, Mitsubishi Tanabe\u00a0Pharma, \u200b\u200bAB Science, Neuralstem, BrainStorm\u00a0Therapeutics, Neuraltus Pharmaceuticals, Biogen, Ionis Pharmaceuticals.<\/li>\n<li><b>Key drugs:\u00a0<\/b>Tirasemtiv, edaravone, masitinib, riluzole, NSI-566, NurOwn, NP-001.<\/li>\n<\/ul>\n","protected":false},"template":"","class_list":["post-390967","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-amyotrophic-lateral-sclerosis","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\/390967","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\/390967\/revisions"}],"predecessor-version":[{"id":576838,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390967\/revisions\/576838"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390967"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}