{"id":12481,"date":"2025-08-31T18:41:52","date_gmt":"2025-08-31T16:41:52","guid":{"rendered":"https:\/\/www.afaf.asso.fr\/?p=12481"},"modified":"2025-08-31T18:41:53","modified_gmt":"2025-08-31T16:41:53","slug":"actualite-vatiquinone","status":"publish","type":"post","link":"https:\/\/www.afaf.asso.fr\/2025\/08\/31\/actualite-vatiquinone\/","title":{"rendered":"Actualit\u00e9 Vatiquinone"},"content":{"rendered":"\n<p class=\"wp-block-paragraph\">La vatiquinone, d\u00e9velopp\u00e9e par <em>PTC Therapeutics<\/em>, \u00a0est une petite mol\u00e9cule, inhibiteur s\u00e9lectif de la 15-lipoxyg\u00e9nase (15-LO), une enzyme qui est un r\u00e9gulateur cl\u00e9 des voies \u00e9nerg\u00e9tiques et du stress oxydatif qui sont perturb\u00e9s dans l&#8217;ataxie de Friedreich.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Dans les suites de la r\u00e9alisation d\u2019un \u00e9tude de phase II\/III ( \u00e9tude MOVE-FA)<em> PTC Therapeutics <\/em>a demand\u00e9 l\u2019autorisation de mise sur le march\u00e9 aupr\u00e8s de la FDA ( Food and Drug Administration). \u00a0<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">L&#8217;essai de phase III MOVE-FA est une \u00e9tude de 18 mois, contr\u00f4l\u00e9e par placebo, qui \u00e9value la vatiquinone par rapport \u00e0 un placebo chez les enfants et les jeunes adultes atteints d&#8217;AF. Le crit\u00e8re d&#8217;\u00e9valuation principal est la variation par rapport \u00e0 la valeur initiale du score mFARS, les crit\u00e8res d&#8217;\u00e9valuation secondaires cl\u00e9s portant sur la marche et les activit\u00e9s de la vie quotidienne. L&#8217;essai MOVE-FA, n&#8217;a pas atteint son crit\u00e8re d&#8217;\u00e9valuation principal, \u00e0 savoir une variation statistiquement significative du score mFARS \u00e0 72 semaines dans la population d&#8217;analyse principale (tous les patients recrut\u00e9s, \u00e2g\u00e9s de 7 \u00e0 21 ans, N = 123), mais a montr\u00e9 des tendances positives dans les crit\u00e8res d&#8217;\u00e9valuation secondaires tels que la stabilit\u00e9 en position debout.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Le 19 ao\u00fbt 2025, PTC Therapeutics, Inc. a annonc\u00e9 que la FDA avait rejet\u00e9 la demande d&#8217;autorisation de mise sur le march\u00e9 (NDA) de <em>PTC Therapeutics<\/em> pour la vatiquinone, invoquant des preuves d&#8217;efficacit\u00e9 insuffisantes et exigeant une \u00e9tude suppl\u00e9mentaire avant une nouvelle soumission.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>La vatiquinone, d\u00e9velopp\u00e9e par PTC Therapeutics, \u00a0est une petite mol\u00e9cule, inhibiteur s\u00e9lectif de la 15-lipoxyg\u00e9nase (15-LO), une enzyme qui est un r\u00e9gulateur cl\u00e9 des voies \u00e9nerg\u00e9tiques et du stress oxydatif qui sont perturb\u00e9s dans l&#8217;ataxie de Friedreich. Dans les suites de la r\u00e9alisation d\u2019un \u00e9tude de phase II\/III ( \u00e9tude MOVE-FA) PTC Therapeutics a demand\u00e9 [&hellip;]<\/p>\n","protected":false},"author":8,"featured_media":7805,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_monsterinsights_skip_tracking":false,"ocean_front_end_style_editor":"no","ocean_post_layout":"","ocean_both_sidebars_style":"","ocean_both_sidebars_content_width":0,"ocean_both_sidebars_sidebars_width":0,"ocean_sidebar":"0","ocean_second_sidebar":"0","ocean_disable_margins":"enable","ocean_add_body_class":"","ocean_shortcode_before_top_bar":"","ocean_shortcode_after_top_bar":"","ocean_shortcode_before_header":"","ocean_shortcode_after_header":"","ocean_has_shortcode":"","ocean_shortcode_after_title":"","ocean_shortcode_before_footer_widgets":"","ocean_shortcode_after_footer_widgets":"","ocean_shortcode_before_footer_bottom":"","ocean_shortcode_after_footer_bottom":"","ocean_display_top_bar":"default","ocean_display_header":"default","ocean_header_style":"","ocean_center_header_left_menu":"0","ocean_custom_header_template":"0","ocean_custom_logo":0,"ocean_custom_retina_logo":0,"ocean_custom_logo_max_width":0,"ocean_custom_logo_tablet_max_width":0,"ocean_custom_logo_mobile_max_width":0,"ocean_custom_logo_max_height":0,"ocean_custom_logo_tablet_max_height":0,"ocean_custom_logo_mobile_max_height":0,"ocean_header_custom_menu":"0","ocean_menu_typo_font_family":"0","ocean_menu_typo_font_subset":"","ocean_menu_typo_font_size":0,"ocean_menu_typo_font_size_tablet":0,"ocean_menu_typo_font_size_mobile":0,"ocean_menu_typo_font_size_unit":"px","ocean_menu_typo_font_weight":"","ocean_menu_typo_font_weight_tablet":"","ocean_menu_typo_font_weight_mobile":"","ocean_menu_typo_transform":"","ocean_menu_typo_transform_tablet":"","ocean_menu_typo_transform_mobile":"","ocean_menu_typo_line_height":0,"ocean_menu_typo_line_height_tablet":0,"ocean_menu_typo_line_height_mobile":0,"ocean_menu_typo_line_height_unit":"","ocean_menu_typo_spacing":0,"ocean_menu_typo_spacing_tablet":0,"ocean_menu_typo_spacing_mobile":0,"ocean_menu_typo_spacing_unit":"","ocean_menu_link_color":"","ocean_menu_link_color_hover":"","ocean_menu_link_color_active":"","ocean_menu_link_background":"","ocean_menu_link_hover_background":"","ocean_menu_link_active_background":"","ocean_menu_social_links_bg":"","ocean_menu_social_hover_links_bg":"","ocean_menu_social_links_color":"","ocean_menu_social_hover_links_color":"","ocean_disable_title":"default","ocean_disable_heading":"default","ocean_post_title":"","ocean_post_subheading":"","ocean_post_title_style":"","ocean_post_title_background_color":"","ocean_post_title_background":0,"ocean_post_title_bg_image_position":"","ocean_post_title_bg_image_attachment":"","ocean_post_title_bg_image_repeat":"","ocean_post_title_bg_image_size":"","ocean_post_title_height":0,"ocean_post_title_bg_overlay":0.5,"ocean_post_title_bg_overlay_color":"","ocean_disable_breadcrumbs":"default","ocean_breadcrumbs_color":"","ocean_breadcrumbs_separator_color":"","ocean_breadcrumbs_links_color":"","ocean_breadcrumbs_links_hover_color":"","ocean_display_footer_widgets":"default","ocean_display_footer_bottom":"default","ocean_custom_footer_template":"0","ocean_post_oembed":"","ocean_post_self_hosted_media":"","ocean_post_video_embed":"","ocean_link_format":"","ocean_link_format_target":"self","ocean_quote_format":"","ocean_quote_format_link":"post","ocean_gallery_link_images":"on","ocean_gallery_id":[],"footnotes":""},"categories":[43],"tags":[],"class_list":["post-12481","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-informations_recherche","entry","has-media","owp-thumbs-layout-horizontal","owp-btn-normal","owp-tabs-layout-section","has-no-thumbnails"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Actualit\u00e9 Vatiquinone - Association Fran\u00e7aise Ataxie de Friedreich<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.afaf.asso.fr\/2025\/08\/31\/actualite-vatiquinone\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Actualit\u00e9 Vatiquinone - Association Fran\u00e7aise Ataxie de Friedreich\" \/>\n<meta property=\"og:description\" content=\"La vatiquinone, d\u00e9velopp\u00e9e par PTC Therapeutics, \u00a0est une petite mol\u00e9cule, inhibiteur s\u00e9lectif de la 15-lipoxyg\u00e9nase (15-LO), une enzyme qui est un r\u00e9gulateur cl\u00e9 des voies \u00e9nerg\u00e9tiques et du stress oxydatif qui sont perturb\u00e9s dans l&#8217;ataxie de Friedreich. Dans les suites de la r\u00e9alisation d\u2019un \u00e9tude de phase II\/III ( \u00e9tude MOVE-FA) PTC Therapeutics a demand\u00e9 [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.afaf.asso.fr\/2025\/08\/31\/actualite-vatiquinone\/\" \/>\n<meta property=\"og:site_name\" content=\"Association Fran\u00e7aise Ataxie de Friedreich\" \/>\n<meta property=\"article:published_time\" content=\"2025-08-31T16:41:52+00:00\" \/>\n<meta property=\"article:modified_time\" content=\"2025-08-31T16:41:53+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.afaf.asso.fr\/wp-content\/uploads\/2022\/12\/NEWS_actualite-scientifiques-e1741791418735.png\" \/>\n\t<meta property=\"og:image:width\" content=\"3552\" \/>\n\t<meta property=\"og:image:height\" content=\"2362\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"author\" content=\"Caroline Gaboriau\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"\u00c9crit par\" \/>\n\t<meta name=\"twitter:data1\" content=\"Caroline Gaboriau\" \/>\n\t<meta name=\"twitter:label2\" content=\"Dur\u00e9e de lecture estim\u00e9e\" \/>\n\t<meta name=\"twitter:data2\" content=\"2 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/2025\\\/08\\\/31\\\/actualite-vatiquinone\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/2025\\\/08\\\/31\\\/actualite-vatiquinone\\\/\"},\"author\":{\"name\":\"Caroline Gaboriau\",\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/#\\\/schema\\\/person\\\/7366aa03c1779ab28140e42231ed7b28\"},\"headline\":\"Actualit\u00e9 Vatiquinone\",\"datePublished\":\"2025-08-31T16:41:52+00:00\",\"dateModified\":\"2025-08-31T16:41:53+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/2025\\\/08\\\/31\\\/actualite-vatiquinone\\\/\"},\"wordCount\":258,\"publisher\":{\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/#organization\"},\"image\":{\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/2025\\\/08\\\/31\\\/actualite-vatiquinone\\\/#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/www.afaf.asso.fr\\\/wp-content\\\/uploads\\\/2022\\\/12\\\/NEWS_actualite-scientifiques-e1741791418735.png\",\"articleSection\":[\"Informations sur la recherche\"],\"inLanguage\":\"fr-FR\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.afaf.asso.fr\\\/2025\\\/08\\\/31\\\/actualite-vatiquinone\\\/\",\"url\":\"https:\\\/\\\/www.afaf.asso.fr\\\/2025\\\/08\\\/31\\\/actualite-vatiquinone\\\/\",\"name\":\"Actualit\u00e9 Vatiquinone - 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