{"id":21542,"date":"2026-10-20T10:19:01","date_gmt":"2026-10-20T10:19:01","guid":{"rendered":"https:\/\/veilles-affaires-sociales.fr\/?p=21542"},"modified":"2026-10-20T10:19:01","modified_gmt":"2026-10-20T10:19:01","slug":"rapport-europeen-public-devaluation-epar-pour-les-medicaments-a-usage-humain-beyonttra-acoramidis-date-dautorisation-10-02-2025-revision-4-statut-autorise-titre-t","status":"publish","type":"post","link":"https:\/\/veilles-affaires-sociales.fr\/?p=21542","title":{"rendered":"Rapport europ\u00e9en public d\u2019\u00e9valuation (EPAR) pour les m\u00e9dicaments \u00e0 usage humain : Beyonttra, acoramidis, Date d\u2019autorisation : 10\/02\/2025, R\u00e9vision : 4, Statut : Autoris\u00e9\u00a0(titre traduit en fran\u00e7ais par l&rsquo;IA)"},"content":{"rendered":"<p>Beyonttra est un m\u00e9dicament destin\u00e9 aux adultes atteints de cardiomyopathie li\u00e9e \u00e0 l\u2019amylose \u00e0 transthyr\u00e9tine (ATTR-CM), qu\u2019il s\u2019agisse de formes variant (avec mutation du g\u00e8ne TTR) ou sauvage (sans mutation identifi\u00e9e). Dans cette maladie, la prot\u00e9ine TTR se d\u00e9stabilise, se fragmente en unit\u00e9s qui se replient anormalement et forment des d\u00e9p\u00f4ts amylo\u00efdes dans le c\u0153ur, entra\u00eenant un durcissement du muscle cardiaque et une alt\u00e9ration de sa capacit\u00e9 de pompage.<\/p>\n<p>Beyonttra contient de l\u2019acoramidis, substance active qui stabilise la prot\u00e9ine TTR en cr\u00e9ant des liaisons fortes avec certaines de ses parties, ce qui limite sa d\u00e9sagr\u00e9gation et vise \u00e0 ralentir la progression de l\u2019ATTR-CM. Le m\u00e9dicament est disponible sous forme de comprim\u00e9s \u00e0 prendre par voie orale deux fois par jour. Il est soumis \u00e0 prescription m\u00e9dicale, et l\u2019instauration du traitement doit \u00eatre r\u00e9alis\u00e9e par un m\u00e9decin exp\u00e9riment\u00e9 dans la prise en charge de l\u2019ATTR-CM. Les informations d\u00e9taill\u00e9es sur l\u2019utilisation, les effets ind\u00e9sirables et les restrictions figurent dans la notice destin\u00e9e aux patients et peuvent \u00eatre pr\u00e9cis\u00e9es par le m\u00e9decin ou le pharmacien.<\/p>\n<p>L\u2019efficacit\u00e9 de Beyonttra a \u00e9t\u00e9 \u00e9valu\u00e9e dans une \u00e9tude principale incluant 632 patients pr\u00e9sentant une ATTR-CM, de type variant ou sauvage, avec sympt\u00f4mes d\u2019insuffisance cardiaque. Le m\u00e9dicament a \u00e9t\u00e9 compar\u00e9 \u00e0 un placebo \u00e0 l\u2019aide d\u2019un crit\u00e8re composite fond\u00e9 sur quatre \u00e9l\u00e9ments : mortalit\u00e9 toutes causes confondues, nombre d\u2019hospitalisations li\u00e9es au c\u0153ur ou \u00e0 la circulation sanguine, \u00e9volution du taux de NT-proBNP (marqueur biologique de stress cardiaque), et variation de la distance parcourue en six minutes de marche. L\u2019\u00e9valuation a utilis\u00e9 un \u00ab win ratio \u00bb int\u00e9grant ces quatre param\u00e8tres, un ratio plus \u00e9lev\u00e9 indiquant un b\u00e9n\u00e9fice plus important du traitement.<\/p>\n<p>Apr\u00e8s 30 mois de traitement, le win ratio en faveur de Beyonttra \u00e9tait d\u2019environ 1,77, ce qui correspond \u00e0 une probabilit\u00e9 environ 77 % plus \u00e9lev\u00e9e de b\u00e9n\u00e9fice clinique par rapport au placebo, selon ce mode de calcul. Sur la m\u00eame p\u00e9riode, le taux de mortalit\u00e9 globale \u00e9tait de 19,3 % dans le groupe Beyonttra, contre 25,7 % dans le groupe placebo, et la distance moyenne de marche en six minutes atteignait 366 m\u00e8tres sous Beyonttra, contre 322 m\u00e8tres sous placebo.<\/p>\n<p>Les effets ind\u00e9sirables les plus fr\u00e9quents (plus de 1 patient sur 10) sont la diarrh\u00e9e et la goutte, cette derni\u00e8re \u00e9tant li\u00e9e \u00e0 une \u00e9l\u00e9vation de l\u2019acide urique dans le sang, pouvant provoquer notamment des inflammations articulaires douloureuses. Aucun autre d\u00e9tail chiffr\u00e9 sur la tol\u00e9rance ou des restrictions d\u2019utilisation sp\u00e9cifiques n\u2019est fourni dans la source, au-del\u00e0 de la n\u00e9cessit\u00e9 de se r\u00e9f\u00e9rer \u00e0 la notice. Aucun \u00e9l\u00e9ment n\u2019est mentionn\u00e9 concernant un pays particulier, une autorit\u00e9 de sant\u00e9 nationale ou europ\u00e9enne, ni d\u2019\u00e9ventuelles conditions d\u2019acc\u00e8s, de prix ou de prise en charge. (contenu r\u00e9sum\u00e9 en fran\u00e7ais par l&rsquo;IA)\u00a0\u200b<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/beyonttra\" target=\"_blank\" class=\"feedzy-rss-link-icon\">Lire la Suite<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Beyonttra est un m\u00e9dicament destin\u00e9 aux adultes atteints de cardiomyopathie li\u00e9e \u00e0 l\u2019amylose \u00e0 transthyr\u00e9tine (ATTR-CM), qu\u2019il s\u2019agisse de formes variant (avec mutation du g\u00e8ne TTR) ou sauvage (sans mutation [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":17698,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center 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