Le document pr\u00e9sente l\u2019\u00e9valuation europ\u00e9enne du m\u00e9dicament Strimvelis par l\u2019Agence europ\u00e9enne des m\u00e9dicaments (EMA) et les conditions d\u2019autorisation et d\u2019utilisation de ce traitement dans l\u2019Union europ\u00e9enne. Strimvelis est un m\u00e9dicament de th\u00e9rapie g\u00e9nique class\u00e9 comme m\u00e9dicament de th\u00e9rapie innovante (\u00ab gene therapy product \u00bb) et comme m\u00e9dicament orphelin depuis le 26 ao\u00fbt 2005, en raison de la raret\u00e9 de la maladie cibl\u00e9e.<\/p>\n
Strimvelis est indiqu\u00e9 dans le traitement du d\u00e9ficit immunitaire combin\u00e9 s\u00e9v\u00e8re d\u00fb \u00e0 un d\u00e9ficit en ad\u00e9nosine d\u00e9saminase (ADA-SCID), une maladie g\u00e9n\u00e9tique rare caract\u00e9ris\u00e9e par une mutation du g\u00e8ne codant l\u2019enzyme ADA. L\u2019absence d\u2019ADA entra\u00eene un dysfonctionnement profond du syst\u00e8me immunitaire, les patients ne survivant en g\u00e9n\u00e9ral pas au-del\u00e0 de deux ans sans traitement efficace. Le m\u00e9dicament est destin\u00e9 aux patients ADA-SCID pour lesquels une greffe de moelle osseuse n\u2019est pas possible faute de donneur apparent\u00e9 compatible.<\/p>\n
Strimvelis est un traitement autologue \u00e0 partir de cellules du patient lui-m\u00eame. Deux pr\u00e9l\u00e8vements de moelle osseuse sont effectu\u00e9s : l\u2019un sert \u00e0 fabriquer le m\u00e9dicament, l\u2019autre est conserv\u00e9 en secours si Strimvelis ne peut pas \u00eatre administr\u00e9 ou ne fonctionne pas. \u00c0 partir de la moelle, les cellules CD34+ (pr\u00e9curseurs pouvant produire des lymphocytes) sont isol\u00e9es. Un g\u00e8ne fonctionnel de l\u2019ADA est ins\u00e9r\u00e9 dans ces cellules au moyen d\u2019un r\u00e9trovirus modifi\u00e9 g\u00e9n\u00e9tiquement, utilis\u00e9 comme vecteur et rendu non pathog\u00e8ne pour l\u2019\u00eatre humain. Le produit ainsi obtenu ne peut \u00eatre utilis\u00e9 que pour le patient dont la moelle a \u00e9t\u00e9 pr\u00e9lev\u00e9e.<\/p>\n
Le traitement ne peut \u00eatre administr\u00e9 que sur prescription, dans un centre de transplantation sp\u00e9cialis\u00e9, par un m\u00e9decin exp\u00e9riment\u00e9 dans la prise en charge de l\u2019ADA-SCID et l\u2019utilisation de ce type de th\u00e9rapie g\u00e9nique. Strimvelis est administr\u00e9 par perfusion intraveineuse d\u2019environ 20 minutes, avec une posologie adapt\u00e9e au poids corporel. Avant l\u2019infusion, une chimioth\u00e9rapie de conditionnement par busulfan est r\u00e9alis\u00e9e afin d\u2019\u00e9liminer les cellules m\u00e9dullaires anormales, et une injection d\u2019antihistaminique est administr\u00e9e pour r\u00e9duire le risque de r\u00e9actions allergiques.<\/p>\n
L\u2019EMA pr\u00e9cise que ce rapport d\u2019\u00e9valuation publique europ\u00e9en n\u2019a pas vocation \u00e0 fournir des conseils pratiques d\u2019utilisation, ceux-ci relevant de la notice destin\u00e9e aux patients ou de l\u2019avis du m\u00e9decin ou du pharmacien. Les \u00e9l\u00e9ments pr\u00e9sent\u00e9s sont principalement r\u00e9glementaires et cliniques, dans le cadre de l\u2019autorisation de mise sur le march\u00e9 dans l\u2019UE d\u2019un traitement innovant pour une maladie rare, avec des conditions strictes de prescription et d\u2019administration dans des structures hautement sp\u00e9cialis\u00e9es. (contenu r\u00e9sum\u00e9 en fran\u00e7ais par l’IA)\u00a0\u200bLire la Suite<\/a><\/p>","protected":false},"excerpt":{"rendered":" Le document pr\u00e9sente l\u2019\u00e9valuation europ\u00e9enne du m\u00e9dicament Strimvelis par l\u2019Agence europ\u00e9enne des m\u00e9dicaments (EMA) et les conditions d\u2019autorisation et d\u2019utilisation de ce traitement dans l\u2019Union europ\u00e9enne. Strimvelis est un m\u00e9dicament […]<\/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 center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[7],"tags":[257,227,226],"class_list":["post-21246","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-medicaments-et-produits-de-sante","tag-ema","tag-ia","tag-sources-etrangeres"],"_links":{"self":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/posts\/21246","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=21246"}],"version-history":[{"count":0,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/posts\/21246\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=\/wp\/v2\/media\/17698"}],"wp:attachment":[{"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=21246"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=21246"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/veilles-affaires-sociales.fr\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=21246"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}