{"id":250738,"date":"2022-11-08T00:00:00","date_gmt":"2022-11-08T00:00:00","guid":{"rendered":"https:\/\/clarivate.com\/products\/research-reports\/report\/nrlfcg0004-2022-biopharma-spinal-muscular-atrophy-landscape-forecast\/"},"modified":"2026-05-12T11:28:34","modified_gmt":"2026-05-12T11:28:34","slug":"nrlfcg0004-2022-biopharma-spinal-muscular-atrophy-landscape-forecast-disease-landscape-forecast","status":"publish","type":"report","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/report\/nrlfcg0004-2022-biopharma-spinal-muscular-atrophy-landscape-forecast-disease-landscape-forecast\/","title":{"rendered":"Spinal Muscular Atrophy &#8211; Landscape &#038; Forecast &#8211; Disease Landscape &#038; Forecast"},"content":{"rendered":"<p>Spinal muscular atrophy (<abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr>) is a neuromuscular disease characterized by progressive muscle weakness; it is a leading genetic cause of death in infants. <abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr> is divided into four clinical subtypes based on severity and age at symptom onset. Since 2016, three disease-modifying therapies have been approved for <abbr title=\"spinal muscular atrophy\">SMA<\/abbr>: Biogen\u2019s Spinraza (nusinersen), Novartis \/ AveXis\u2019s Zolgensma (formerly AVXS-101; onasemnogene abeparvovec), and Roche \/ Genentech \/ PTC Therapeutics\u2019 Evrysdi (risdiplam). Although these therapies have expanded treatment options for patients, the therapies are associated with many challenges, including high prices, which limit patients\u2019 access to them. In the <abbr title=\"spinal muscular atrophy\">SMA<\/abbr> pipeline are many therapies with diverse mechanisms of action and routes of administration; if approved, they will allow physicians to treat patients based on their symptoms, comorbidities, and preferences. In this content, we will explore the clinical and commercial potential of key current and emerging therapies for <abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr>.<\/p>\n<p><strong>Questions a<\/strong><strong>nswered<\/strong><\/p>\n<ul class=\"round-bullets\">\n<li>How large is the treatable <abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr> population, and how will its size change through 2031?<\/li>\n<li>What is the current state of treatment of <abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr>? Which are the most important drugs and why? What are interviewed experts\u2019 insights on current treatments? What clinical needs remain unfulfilled?<\/li>\n<li>What pipeline products are most promising, and what sales \/ uptake could they secure in <abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr>? What therapies of note are progressing in earlier phases?<\/li>\n<li>What are the drivers and constraints in the <abbr data-abbreviation-entity=\"6515\" title=\"spinal muscular atrophy\">SMA<\/abbr> market, and how will this market evolve over the forecast period?<\/li>\n<\/ul>\n<p><strong>Content highlights<\/strong><\/p>\n<p><strong>Geography:<\/strong> United States and <abbr data-abbreviation-entity=\"6092\" title=\"France, Germany, Italy, Spain, UK\">EU5<\/abbr>.<\/p>\n<p><strong>Primary research:<\/strong> Six country-specific interviews with thought-leading neurologists supported by survey data collected for this study.<\/p>\n<p><strong>Epidemiology:<\/strong> Diagnosed prevalence of <abbr title=\"spinal muscular atrophy\">SMA<\/abbr> by country, segmented by subtype (I-IV).<\/p>\n<p><strong>Forecast:<\/strong> Drug-level sales and patient share of key <abbr title=\"spinal muscular atrophy\">SMA<\/abbr> therapies through 2031.<\/p>\n<p><strong>Emerging therapies:<\/strong> Phase III\/<abbr data-abbreviation-entity=\"5086\" title=\"preregistered\">PR<\/abbr>: 3drugs; Phase II: 1drug; coverage of select early-phase products<\/p>\n<p><strong>Product d<\/strong><strong>escription<\/strong><\/p>\n<p><em>Niche &#038; Rare Disease Landscape &#038; Forecast<\/em> provides comprehensive market intelligence with 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<p>This <em>Niche &#038; Rare Disease Landscape &#038; Forecast<\/em> report introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.<\/p>\n","protected":false},"template":"","class_list":["post-250738","report","type-report","status-publish","hentry","report-gateway-biopharma","biopharma-therapy-areas-neurology","biopharma-therapy-areas-spinal-muscular-atrophy","biopharma-geography-us","biopharma-date-960"],"acf":[],"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/250738","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":10,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/250738\/revisions"}],"predecessor-version":[{"id":576194,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/report\/250738\/revisions\/576194"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=250738"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}