{"id":6238,"date":"2018-02-20T11:16:14","date_gmt":"2018-02-20T11:16:14","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=6238"},"modified":"2022-05-03T18:29:13","modified_gmt":"2022-05-03T18:29:13","slug":"decision-fda-office-ataluren","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/","title":{"rendered":"Decision from the FDA&#8217;s Office for Ataluren"},"content":{"rendered":"<p><strong>Today, <b>PTC Therapeutics, Inc, <\/b>announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA&#8217;s prior position and denied PTC&#8217;s appeal of the Complete Response Letter concerning the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. <\/strong><\/p>\n<p><strong>This would involve a re-submission of an NDA containing the current data on the effectiveness of ataluren with new data to be generated on dystrophin production in nonsense mutation Duchenne muscular dystrophy (nmDMD) patients&#8217; muscles, as quantified by procedures to be agreed upon between PTC and the FDA and using newer technologies. The letter adds that PTC&#8217;s Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework. In a clarification teleconference with the FDA promptly after receiving the letter, PTC indicated its intent to follow the FDA&#8217;s recommendation and preliminarily discussed methods to collect such dystrophin data and expedite this potential path forward.<\/strong><\/p>\n<h4><\/h4>\n<h4><strong>The patient will still have access to treatment:<\/strong><\/h4>\n<p>Based on these interactions, PTC currently intends to maintain patients in the U.S. currently receiving ataluren for nmDMD through an expanded access clinical program during this process.<\/p>\n<h4>About the AdCom watch our video:<\/h4>\n<p><iframe loading=\"lazy\" title=\"FDA Advisory Committee (AdCom) hears the voice of the DMD community\" width=\"1140\" height=\"641\" src=\"https:\/\/www.youtube.com\/embed\/J3qcd2aNadI?start=66&#038;feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture\" allowfullscreen><\/iframe><\/p>\n<p>&nbsp;<\/p>\n<h4><strong>About Translarna\u2122 (ataluren)<\/strong><\/h4>\n<p>This treatment is the first to target an underlying genetic cause of DMD: a nonsense mutation (nmDMD). It aims to slow the progression of DMD by restoring the function of a specific protein, dystrophin. In DMD, a nonsense mutation corrupts the genetic machinery that builds a working version of this protein, which is essential for proper neuromuscular function.<\/p>\n<p>In clinical trials, ataluren has shown some benefit in slowing the loss of motor skills in some children with nmDMD. Some boys treated with ataluren could more easily undertake a range of physical activities, including walking, climbing and descending stairs and other motor functions.<\/p>\n<p><strong>In 2014<\/strong>, the European Medicines Agency granted Translarna\u2122 (ataluren) conditional marketing authorization to treat nmDMD in children of 5 years of age or more and still able to walk. Translarna\u2122 (ataluren) is also approved for use in South Korea and Israel.<\/p>\n<p class=\"p1\"><span class=\"s1\"><strong>At the end of 2016<\/strong>, Translarna\u2122 (ataluren) received marketing authorization by the European Medicines Agency and is available in Europe and regions that reference that authorization.<\/span><\/p>\n<p><strong>On the 25h of October<\/strong>, the Office of Drug Evaluation I of the FDA provided a Complete Response Letter (CRL) for the application to market ataluren in the United States. This means that the FDA believes ataluren cannot be approved at this time. PTC Therapeutics Inc. is planning on filing a formal dispute resolution request next week. The dispute resolution process gives PTC an opportunity to appeal the letter issued by the Neurology division and have the decision on the application reviewed by the Office of New Products within the FDA. <a href=\"http:\/\/ir.ptcbio.com\/releaseDetail.cfm?ReleaseID=1045384\" rel=\"noopener noreferrer\">PTC\u00a0Press Release<\/a><\/p>\n<p>&nbsp;<\/p>\n<h4>Understanding Ataluren approbation process<\/h4>\n<p><strong>For more information<\/strong><\/p>\n<p><a href=\"\/?p=5432\" rel=\"noopener\">What is the AdCom?<\/a><br \/>\n<a href=\"\/?p=5536\" rel=\"noopener\">Families at the AdCom September 2017<\/a><br \/>\n<a href=\"\/?p=5765\" rel=\"noopener\">FDA close the door on Ataluren<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Today, PTC Therapeutics, Inc, announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA&#8217;s prior position and denied PTC&#8217;s appeal of the Complete Response Letter concerning the New Drug Application (NDA) for ataluren. In its letter, the Office of New Drugs recommended a possible path forward for [&hellip;]<\/p>\n","protected":false},"author":3,"featured_media":6248,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[12],"tags":[],"class_list":["post-6238","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Decision from the FDA&#039;s Office for ataluren<\/title>\n<meta name=\"description\" content=\"the FDA&#039;s prior position and denied PTC&#039;s appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Decision from the FDA&#039;s Office for ataluren\" \/>\n<meta property=\"og:description\" content=\"the FDA&#039;s prior position and denied PTC&#039;s appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/\" \/>\n<meta property=\"og:site_name\" content=\"La Force dmd\" \/>\n<meta property=\"article:published_time\" content=\"2018-02-20T11:16:14+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2022-05-03T18:29:13+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/laforcedmd.com\/wp-content\/uploads\/2018\/02\/decision-fda-office-ataluren.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"815\" \/>\n\t<meta property=\"og:image:height\" content=\"356\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"La Force DMD\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"La Force DMD\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"3 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/\"},\"author\":{\"name\":\"La Force DMD\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#\\\/schema\\\/person\\\/c7bd653b3f3d7fa23ef1721673d744cb\"},\"headline\":\"Decision from the FDA&#8217;s Office for Ataluren\",\"datePublished\":\"2018-02-20T11:16:14+00:00\",\"dateModified\":\"2022-05-03T18:29:13+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/\"},\"wordCount\":527,\"commentCount\":0,\"publisher\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#organization\"},\"image\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/laforcedmd.com\\\/wp-content\\\/uploads\\\/2018\\\/02\\\/decision-fda-office-ataluren.jpg\",\"articleSection\":[\"News\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/\",\"url\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/\",\"name\":\"Decision from the FDA's Office for ataluren\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#primaryimage\"},\"image\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/laforcedmd.com\\\/wp-content\\\/uploads\\\/2018\\\/02\\\/decision-fda-office-ataluren.jpg\",\"datePublished\":\"2018-02-20T11:16:14+00:00\",\"dateModified\":\"2022-05-03T18:29:13+00:00\",\"description\":\"the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren.\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#primaryimage\",\"url\":\"https:\\\/\\\/laforcedmd.com\\\/wp-content\\\/uploads\\\/2018\\\/02\\\/decision-fda-office-ataluren.jpg\",\"contentUrl\":\"https:\\\/\\\/laforcedmd.com\\\/wp-content\\\/uploads\\\/2018\\\/02\\\/decision-fda-office-ataluren.jpg\",\"width\":815,\"height\":356,\"caption\":\"The FDA's office decision about the ataluren.\"},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/decision-fda-office-ataluren\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Accueil\",\"item\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Decision from the FDA&#8217;s Office for Ataluren\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#website\",\"url\":\"https:\\\/\\\/laforcedmd.com\\\/\",\"name\":\"La Force dmd\",\"description\":\"Dystrophie musculaire de Duchenne\",\"publisher\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#organization\"},\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/laforcedmd.com\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"},{\"@type\":\"Organization\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#organization\",\"name\":\"La Force dmd\",\"url\":\"https:\\\/\\\/laforcedmd.com\\\/\",\"logo\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#\\\/schema\\\/logo\\\/image\\\/\",\"url\":\"https:\\\/\\\/laforcedmd.com\\\/wp-content\\\/uploads\\\/2022\\\/06\\\/logo-la-force-dmd.jpg\",\"contentUrl\":\"https:\\\/\\\/laforcedmd.com\\\/wp-content\\\/uploads\\\/2022\\\/06\\\/logo-la-force-dmd.jpg\",\"width\":500,\"height\":125,\"caption\":\"La Force dmd\"},\"image\":{\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#\\\/schema\\\/logo\\\/image\\\/\"}},{\"@type\":\"Person\",\"@id\":\"https:\\\/\\\/laforcedmd.com\\\/#\\\/schema\\\/person\\\/c7bd653b3f3d7fa23ef1721673d744cb\",\"name\":\"La Force DMD\",\"image\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/1362e69258784090c887e017c49a0bab8291c910310241733859975cc5ae0c07?s=96&d=mm&r=g\",\"url\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/1362e69258784090c887e017c49a0bab8291c910310241733859975cc5ae0c07?s=96&d=mm&r=g\",\"contentUrl\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/1362e69258784090c887e017c49a0bab8291c910310241733859975cc5ae0c07?s=96&d=mm&r=g\",\"caption\":\"La Force DMD\"},\"url\":\"https:\\\/\\\/laforcedmd.com\\\/en\\\/author\\\/la-force-dmd\\\/\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Decision from the FDA's Office for ataluren","description":"the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren.","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/","og_locale":"en_US","og_type":"article","og_title":"Decision from the FDA's Office for ataluren","og_description":"the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren.","og_url":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/","og_site_name":"La Force dmd","article_published_time":"2018-02-20T11:16:14+00:00","article_modified_time":"2022-05-03T18:29:13+00:00","og_image":[{"width":815,"height":356,"url":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2018\/02\/decision-fda-office-ataluren.jpg","type":"image\/jpeg"}],"author":"La Force DMD","twitter_card":"summary_large_image","twitter_misc":{"Written by":"La Force DMD","Est. reading time":"3 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#article","isPartOf":{"@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/"},"author":{"name":"La Force DMD","@id":"https:\/\/laforcedmd.com\/#\/schema\/person\/c7bd653b3f3d7fa23ef1721673d744cb"},"headline":"Decision from the FDA&#8217;s Office for Ataluren","datePublished":"2018-02-20T11:16:14+00:00","dateModified":"2022-05-03T18:29:13+00:00","mainEntityOfPage":{"@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/"},"wordCount":527,"commentCount":0,"publisher":{"@id":"https:\/\/laforcedmd.com\/#organization"},"image":{"@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#primaryimage"},"thumbnailUrl":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2018\/02\/decision-fda-office-ataluren.jpg","articleSection":["News"],"inLanguage":"en-US"},{"@type":"WebPage","@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/","url":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/","name":"Decision from the FDA's Office for ataluren","isPartOf":{"@id":"https:\/\/laforcedmd.com\/#website"},"primaryImageOfPage":{"@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#primaryimage"},"image":{"@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#primaryimage"},"thumbnailUrl":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2018\/02\/decision-fda-office-ataluren.jpg","datePublished":"2018-02-20T11:16:14+00:00","dateModified":"2022-05-03T18:29:13+00:00","description":"the FDA's prior position and denied PTC's appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren.","breadcrumb":{"@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/"]}]},{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#primaryimage","url":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2018\/02\/decision-fda-office-ataluren.jpg","contentUrl":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2018\/02\/decision-fda-office-ataluren.jpg","width":815,"height":356,"caption":"The FDA's office decision about the ataluren."},{"@type":"BreadcrumbList","@id":"https:\/\/laforcedmd.com\/en\/decision-fda-office-ataluren\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Accueil","item":"https:\/\/laforcedmd.com\/en\/"},{"@type":"ListItem","position":2,"name":"Decision from the FDA&#8217;s Office for Ataluren"}]},{"@type":"WebSite","@id":"https:\/\/laforcedmd.com\/#website","url":"https:\/\/laforcedmd.com\/","name":"La Force dmd","description":"Dystrophie musculaire de Duchenne","publisher":{"@id":"https:\/\/laforcedmd.com\/#organization"},"potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/laforcedmd.com\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"},{"@type":"Organization","@id":"https:\/\/laforcedmd.com\/#organization","name":"La Force dmd","url":"https:\/\/laforcedmd.com\/","logo":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/laforcedmd.com\/#\/schema\/logo\/image\/","url":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2022\/06\/logo-la-force-dmd.jpg","contentUrl":"https:\/\/laforcedmd.com\/wp-content\/uploads\/2022\/06\/logo-la-force-dmd.jpg","width":500,"height":125,"caption":"La Force dmd"},"image":{"@id":"https:\/\/laforcedmd.com\/#\/schema\/logo\/image\/"}},{"@type":"Person","@id":"https:\/\/laforcedmd.com\/#\/schema\/person\/c7bd653b3f3d7fa23ef1721673d744cb","name":"La Force DMD","image":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/secure.gravatar.com\/avatar\/1362e69258784090c887e017c49a0bab8291c910310241733859975cc5ae0c07?s=96&d=mm&r=g","url":"https:\/\/secure.gravatar.com\/avatar\/1362e69258784090c887e017c49a0bab8291c910310241733859975cc5ae0c07?s=96&d=mm&r=g","contentUrl":"https:\/\/secure.gravatar.com\/avatar\/1362e69258784090c887e017c49a0bab8291c910310241733859975cc5ae0c07?s=96&d=mm&r=g","caption":"La Force DMD"},"url":"https:\/\/laforcedmd.com\/en\/author\/la-force-dmd\/"}]}},"_links":{"self":[{"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/posts\/6238","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/comments?post=6238"}],"version-history":[{"count":1,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/posts\/6238\/revisions"}],"predecessor-version":[{"id":11240,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/posts\/6238\/revisions\/11240"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/media\/6248"}],"wp:attachment":[{"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/media?parent=6238"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/categories?post=6238"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/laforcedmd.com\/en\/wp-json\/wp\/v2\/tags?post=6238"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}