{"id":390758,"date":"2017-12-21T00:00:00","date_gmt":"2017-12-21T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2017-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2017\/"},"modified":"2026-03-31T10:49:01","modified_gmt":"2026-03-31T10:49:01","slug":"nrlfcg0003-2017-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2017","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0003-2017-biopharma-muscular-dystrophy-niche-rare-disease-landscape-forecast-us-eu5-2017\/","title":{"rendered":"Muscular Dystrophy | Niche &#038; Rare Disease Landscape &#038; Forecast | US\/EU5 | 2017"},"content":{"rendered":"<p>Muscular\u00a0dystrophies\u00a0(<abbr data-original-title=\"muscular dystrophy\" title=\"\">MD<\/abbr>s) are a spectrum of genetic disorders characterized by muscle weakness that in severe disease forms can lead to loss of ambulation and early death.\u00a0<abbr data-original-title=\"muscular dystrophy\" title=\"\">MD<\/abbr>s share common histological features such as reduction in muscle fiber size, degeneration, and presence of connective and adipose tissue instead of muscle.\u00a0Duchenne\u00a0muscular dystrophy (<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>) is the most common subtype with childhood onset, while\u00a0myotonicdystrophy is the most common in adults. The unmet need for effective treatments that can meaningfully delay or halt progressive muscle degeneration in\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>, as well as in other severe and moderately severe\u00a0<abbr data-original-title=\"muscular dystrophy\" title=\"\">MD<\/abbr>s, is high.\u00a0Ataluren\u00a0(PTC Therapeutics\u2019\u00a0Translarna) targets a genetically defined subset of\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>\u00a0patients and has been available in some markets following its 2014 European approval;\u00a0eteplirsen\u00a0(Sarepta\u2019s\u00a0Exondys\u00a051) was granted conditional approval in the United States in 2016 and targets a different group of\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>\u00a0patients who share a similar genetic mutation at the basis of their disease. Though both drugs target the underlying cause of the disease and thus could potentially modify its course, their efficacy as observed in clinical trials thus far appears modest, and the unmet need for effective disease-modifying therapies that would provide relief to all\u00a0<abbr data-original-title=\"muscular dystrophy\" title=\"\">MD<\/abbr>\u00a0patients remains high.<\/p>\n<p><u><strong>Questions Answered:<\/strong><\/u><\/p>\n<ul>\n<li>How large is the diagnosed\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>\u00a0population in the United States and\u00a0<abbr data-original-title=\"France, Germany, Italy, Spain, UK\" title=\"\">EU5<\/abbr>, and how will its size change over time?<\/li>\n<li>What is the current treatment landscape for\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>\u00a0patients, and how will it change in the next ten years?<\/li>\n<li>What is the market share of current agents in the U.S.\/EU5 DMD market?<\/li>\n<li>What do interviewed experts believe is the greatest unmet medical need in the treatment of\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>?<\/li>\n<li>What are interviewed experts\u2019 insights on emerging therapies?<\/li>\n<li>How will emerging therapies fit into future medical practice?<\/li>\n<\/ul>\n<p><u><strong>Scope:<\/strong><\/u><\/p>\n<ul>\n<li><strong>Markets covered:<\/strong>\u00a0United States, France, Germany, Italy, Spain, and the United Kingdom.<\/li>\n<li><strong>Primary research:<\/strong>\u00a0Six country-specific interviews with thought-leading pediatric neurologists and pediatric cardiologists.<\/li>\n<li><strong>Epidemiology:<\/strong>\u00a0Diagnosed prevalence of\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>\u00a0patients and drug-treated patients by country segmented by exon skipping pattern and by ambulatory status for the base year 2016 and out years 2021 and 2026. Diagnosed prevalence of Becker muscular dystrophy (<abbr data-original-title=\"Becker muscular dystrophy\" title=\"\">BMD<\/abbr>) and limb-girdle muscular dystrophy (<abbr data-original-title=\"limb-girdle muscular dystrophy\" title=\"\">LGMD<\/abbr>).<\/li>\n<li><strong>Market forecast:<\/strong>\u00a0Drug-level sales and patient share of key\u00a0<abbr data-original-title=\"Duchenne muscular dystrophy\" title=\"\">DMD<\/abbr>\u00a0therapies in 2016 and 2026.<\/li>\n<li><strong>Emerging therapies:<\/strong>\u00a0Phase III\/<abbr data-original-title=\"preregistered\" title=\"\">PR<\/abbr>: 4 drugs, Phase II: 6; coverage of select Phase I\/II and I products.<\/li>\n<li><strong>Key companies:\u00a0<\/strong>AMO Pharma, aTyr Pharma, Akashi Therapeutics, Biogen, Catabasis Pharmaceuticals, EspeRare Foundation, FibroGen, Italfarmaco SpA, Milo Biotechnology, Pfizer, PTC Therapeutics, ReveraGen BioPharma, Roche, Santhera Pharmaceuticals, Sarepta Therapeutics.<\/li>\n<li><strong>Key drugs:<\/strong>\u00a0AAV1-FS344\u200b, ataluren (PTC Therapeutics\u2019 Translarna),\u00a0<abbr data-original-title=\"Bristol-Myers Squibb\" title=\"\">BMS<\/abbr>-986089, deflazacort (PTC Therapeutics\u2019 Emflaza, generics), domagrozumab, edasalonexent, eteplirsen (Sarepta&#8217;s Exondys 51), ezutromid, givinostat, golodirsen, halofuginone delayed release, idebenone, omigapil\u200b, pamrevlumab\u200b, prednisone (generics), rAAVrh74.MCK.micro-Dystrophin, resolaris, rimeporide\u200b, SRP-4045, tideglusib, vamorolone\u200b.<\/li>\n<\/ul>\n","protected":false},"template":"","class_list":["post-390758","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-muscular-dystrophy","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\/390758","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\/390758\/revisions"}],"predecessor-version":[{"id":576761,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/390758\/revisions\/576761"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=390758"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}