{"id":111332,"date":"2020-08-14T06:23:14","date_gmt":"2020-08-14T13:23:14","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2020\/08\/fda-approves-viltolarsen-for-duchenne-muscular-dystrophy"},"modified":"2020-08-14T06:23:14","modified_gmt":"2020-08-14T13:23:14","slug":"fda-approves-viltolarsen-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2020\/08\/fda-approves-viltolarsen-for-duchenne-muscular-dystrophy","title":{"rendered":"FDA Approves Viltolarsen for Duchenne Muscular Dystrophy"},"content":{"rendered":"<p style=\"padding-right: 20px\"><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/fda-approves-viltolarsen-for-duchenne-muscular-dystrophy2.jpg\"><\/a><\/p>\n<p>The US Food and Drug Administration (FDA) has approved viltolarsen (Viltepso; NS Pharma) for the treatment of patients with <a href=\"https:\/\/www.hcplive.com\/clinical\/duchenne-muscular-dystrophy\" target=\"_blank\"> Duchenne muscular dystrophy<\/a> amenable to exon 53 skipping, making it only the second FDA-approved therapy for this specific <em>DMD<\/em> gene mutation.<\/p>\n<p>The agent from NS Pharma, delivered via weekly intravenous infusion, was granted accelerated approval via its priority review, fast track, orphan drug, and rare disease designations after its <a href=\"https:\/\/www.neurologylive.com\/clinical-focus\/viltolarsen-nda-accepted-for-duchenne-muscular-dystrophy-treatment\" target=\"_blank\"> new drug application<\/a> was accepted earlier this year. In March, NS Pharma launched an <a href=\"https:\/\/www.neurologylive.com\/clinical-focus\/viltolarsen-available-through-expanded-access-program--\" target=\"_blank\"> expanded access program<\/a> for qualified patients.<\/p>\n<p>The approval was granted based on findings from a <a href=\"https:\/\/www.neurologylive.com\/clinical-focus\/viltolarsen-induces-de-novo-dystrophin-production-in-duchenne-muscular-dystrophy\" target=\"_blank\"> phase 2 clinical trial<\/a> (NCT02740972) and long-term extension study, details of which were recently published in <em>JAMA Neurology<\/em>. Among 16 participants age 4 to 9, significant drug-induced dystrophin production was observed in both viltolarsen dose cohorts (40 mg\/kg per week: mean, 5.7% [range, 3.2\u201310.3] of normal; 80 mg\/kg per week: mean, 5.9% [range, 1.1\u201314.4] of normal), with 15 (94%) patients achieving dystrophin levels greater than 2% of normal and 14 of 16 (88%) achieving levels greater than 3% of normal.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The US Food and Drug Administration (FDA) has approved viltolarsen (Viltepso; NS Pharma) for the treatment of patients with Duchenne muscular dystrophy amenable to exon 53 skipping, making it only the second FDA-approved therapy for this specific DMD gene mutation. The agent from NS Pharma, delivered via weekly intravenous infusion, was granted accelerated approval via [\u2026]<\/p>\n","protected":false},"author":513,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412],"tags":[],"class_list":["post-111332","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/111332","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/513"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=111332"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/111332\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=111332"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=111332"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=111332"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}