{"id":20015,"date":"2026-10-06T14:06:47","date_gmt":"2026-10-06T14:06:47","guid":{"rendered":"https:\/\/veilles-affaires-sociales.fr\/?p=20015"},"modified":"2026-10-06T14:06:47","modified_gmt":"2026-10-06T14:06:47","slug":"avis-de-lansm-du-18-09-2026-sur-le-medicament-pyrukynd-dans-le-cadre-dune-demande-daap","status":"publish","type":"post","link":"https:\/\/veilles-affaires-sociales.fr\/?p=20015","title":{"rendered":"Avis de l&rsquo;ANSM du 18\/09\/2026 sur le m\u00e9dicament Pyrukynd dans le cadre d&rsquo;une demande d&rsquo;AAP"},"content":{"rendered":"<p>L\u2019Agence nationale de s\u00e9curit\u00e9 du m\u00e9dicament et des produits de sant\u00e9 (ANSM) a rendu le 18 septembre 2026 un avis concernant le m\u00e9dicament Pyrukynd 100 mg, comprim\u00e9s pellicul\u00e9s (DCI : mitapivat), d\u00e9velopp\u00e9 par la soci\u00e9t\u00e9 Avanzanite Bioscience BV. Cet avis intervient dans le cadre d\u2019une demande d\u2019autorisation d\u2019acc\u00e8s pr\u00e9coce (AAP) visant l\u2019indication suivante : traitement de l\u2019an\u00e9mie h\u00e9molytique associ\u00e9e \u00e0 la dr\u00e9panocytose chez les patients adultes.<\/p>\n<p>La demande initiale d\u2019AAP a \u00e9t\u00e9 d\u00e9pos\u00e9e le 12 mai 2026 et a fait l\u2019objet de compl\u00e9ments successifs fournis par le demandeur les 28 mai, 30 juin, 28 ao\u00fbt, 8 septembre, 13 septembre et 18 septembre 2026. Au terme de l\u2019instruction, l\u2019ANSM indique qu\u2019elle ne peut attester de l\u2019existence d\u2019une forte pr\u00e9somption d\u2019efficacit\u00e9 et de s\u00e9curit\u00e9 de Pyrukynd 100 mg dans l\u2019indication revendiqu\u00e9e. La justification scientifique d\u00e9taill\u00e9e de cette position est pr\u00e9sent\u00e9e dans une annexe accessible via la fiche AAP d\u00e9di\u00e9e \u00e0 Pyrukynd.<\/p>\n<p>L\u2019avis d\u00e9favorable sur la forte pr\u00e9somption d\u2019efficacit\u00e9 et de s\u00e9curit\u00e9 a des implications directes sur la possibilit\u00e9 de recourir \u00e0 ce m\u00e9dicament dans le cadre du dispositif d\u2019acc\u00e8s pr\u00e9coce en France pour les adultes atteints de dr\u00e9panocytose avec an\u00e9mie h\u00e9molytique. Aucun autre \u00e9l\u00e9ment chiffr\u00e9 ni comparaison internationale n\u2019est fourni dans le document.\u00a0(contenu r\u00e9sum\u00e9 par l&rsquo;IA)<a href=\"https:\/\/ansm.sante.fr\/actualites\/avis-de-lansm-du-18-09-2026-sur-le-medicament-pyrukynd-dans-le-cadre-dune-demande-daap\" target=\"_blank\" class=\"feedzy-rss-link-icon\">Lire la Suite<\/a>\u200b<\/p>","protected":false},"excerpt":{"rendered":"<p>L\u2019Agence nationale de s\u00e9curit\u00e9 du m\u00e9dicament et des produits de sant\u00e9 (ANSM) a rendu le 18 septembre 2026 un avis concernant le m\u00e9dicament Pyrukynd 100 mg, comprim\u00e9s pellicul\u00e9s (DCI : [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3852,"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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