{"id":37255,"date":"2022-05-06T15:36:08","date_gmt":"2022-05-06T19:36:08","guid":{"rendered":"https:\/\/wfh.org\/biomarin-follows-up-on-previously-reported-serious-adverse-event-in-its-phase-i-ii-gene-therapy-clinical-trial-for-haemophilia-a\/"},"modified":"2024-04-12T09:56:21","modified_gmt":"2024-04-12T13:56:21","slug":"biomarin-fait-suite-a-un-evenement-indesirable-grave-precedemment-signale-dans-son-essai-clinique-de-therapie-genique-de-phase-i-ii-pour-lhemophilie-a","status":"publish","type":"post","link":"https:\/\/wfh.org\/fr\/article\/biomarin-fait-suite-a-un-evenement-indesirable-grave-precedemment-signale-dans-son-essai-clinique-de-therapie-genique-de-phase-i-ii-pour-lhemophilie-a\/","title":{"rendered":"BioMarin fait suite \u00e0 un \u00e9v\u00e9nement ind\u00e9sirable grave pr\u00e9c\u00e9demment signal\u00e9 dans son essai clinique de th\u00e9rapie g\u00e9nique de phase I\/II pour l\u2019h\u00e9mophilie A"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-post\" data-elementor-id=\"37255\" class=\"elementor elementor-37255 elementor-36595\" data-elementor-post-type=\"post\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-ff4eb04 elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"ff4eb04\" data-element_type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-8e72bdb\" data-id=\"8e72bdb\" data-element_type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-7d8df3d gallery-spacing-custom elementor-widget elementor-widget-image-gallery\" data-id=\"7d8df3d\" data-element_type=\"widget\" data-widget_type=\"image-gallery.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-image-gallery .gallery-item{display:inline-block;text-align:center;vertical-align:top;width:100%;max-width:100%;margin:0 auto}.elementor-image-gallery .gallery-item img{margin:0 auto}.elementor-image-gallery .gallery-item .gallery-caption{margin:0}.elementor-image-gallery figure img{display:block}.elementor-image-gallery figure figcaption{width:100%}.gallery-spacing-custom .elementor-image-gallery .gallery-icon{padding:0}@media (min-width:768px){.elementor-image-gallery .gallery-columns-2 .gallery-item{max-width:50%}.elementor-image-gallery .gallery-columns-3 .gallery-item{max-width:33.33%}.elementor-image-gallery .gallery-columns-4 .gallery-item{max-width:25%}.elementor-image-gallery .gallery-columns-5 .gallery-item{max-width:20%}.elementor-image-gallery .gallery-columns-6 .gallery-item{max-width:16.666%}.elementor-image-gallery .gallery-columns-7 .gallery-item{max-width:14.28%}.elementor-image-gallery .gallery-columns-8 .gallery-item{max-width:12.5%}.elementor-image-gallery .gallery-columns-9 .gallery-item{max-width:11.11%}.elementor-image-gallery .gallery-columns-10 .gallery-item{max-width:10%}}@media (min-width:480px) and (max-width:767px){.elementor-image-gallery .gallery.gallery-columns-2 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-3 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-4 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-5 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-6 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-7 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-8 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-9 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-10 .gallery-item{max-width:50%}}@media (max-width:479px){.elementor-image-gallery .gallery.gallery-columns-2 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-3 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-4 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-5 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-6 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-7 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-8 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-9 .gallery-item,.elementor-image-gallery .gallery.gallery-columns-10 .gallery-item{max-width:100%}}<\/style>\t\t<div class=\"elementor-image-gallery\">\n\t\t\t<div id='gallery-1' class='gallery galleryid-37255 gallery-columns-3 gallery-size-full'><figure class='gallery-item'>\n\t\t\t<div class='gallery-icon landscape'>\n\t\t\t\t<img fetchpriority=\"high\" decoding=\"async\" width=\"770\" height=\"350\" src=\"https:\/\/wfh.org\/wp-content\/uploads\/2022\/01\/EHC.png\" class=\"attachment-full size-full\" alt=\"Logo European haemophilia consortium\" srcset=\"https:\/\/wfh.org\/wp-content\/uploads\/2022\/01\/EHC.png 770w, https:\/\/wfh.org\/wp-content\/uploads\/2022\/01\/EHC-300x136.png 300w, https:\/\/wfh.org\/wp-content\/uploads\/2022\/01\/EHC-768x349.png 768w\" sizes=\"(max-width: 770px) 100vw, 770px\" \/>\n\t\t\t<\/div><\/figure><figure class='gallery-item'>\n\t\t\t<div class='gallery-icon landscape'>\n\t\t\t\t<img decoding=\"async\" width=\"600\" height=\"300\" src=\"https:\/\/wfh.org\/wp-content\/uploads\/2021\/12\/NHF-logo.png\" class=\"attachment-full size-full\" alt=\"\" srcset=\"https:\/\/wfh.org\/wp-content\/uploads\/2021\/12\/NHF-logo.png 600w, https:\/\/wfh.org\/wp-content\/uploads\/2021\/12\/NHF-logo-300x150.png 300w\" sizes=\"(max-width: 600px) 100vw, 600px\" \/>\n\t\t\t<\/div><\/figure><figure class='gallery-item'>\n\t\t\t<div class='gallery-icon landscape'>\n\t\t\t\t<img decoding=\"async\" width=\"1277\" height=\"603\" src=\"https:\/\/wfh.org\/wp-content\/uploads\/2021\/04\/WFH-trilingual-logo-EN.png\" class=\"attachment-full size-full\" alt=\"\" aria-describedby=\"gallery-1-29974\" srcset=\"https:\/\/wfh.org\/wp-content\/uploads\/2021\/04\/WFH-trilingual-logo-EN.png 1277w, https:\/\/wfh.org\/wp-content\/uploads\/2021\/04\/WFH-trilingual-logo-EN-192x91.png 192w, https:\/\/wfh.org\/wp-content\/uploads\/2021\/04\/WFH-trilingual-logo-EN-768x363.png 768w\" sizes=\"(max-width: 1277px) 100vw, 1277px\" \/>\n\t\t\t<\/div>\n\t\t\t\t<figcaption class='wp-caption-text gallery-caption' id='gallery-1-29974'>\n\t\t\t\tstock\n\t\t\t\t<\/figcaption><\/figure>\n\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-153242c elementor-widget elementor-widget-text-editor\" data-id=\"153242c\" data-element_type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - v3.23.0 - 25-07-2024 *\/\n.elementor-widget-text-editor.elementor-drop-cap-view-stacked .elementor-drop-cap{background-color:#69727d;color:#fff}.elementor-widget-text-editor.elementor-drop-cap-view-framed .elementor-drop-cap{color:#69727d;border:3px solid;background-color:transparent}.elementor-widget-text-editor:not(.elementor-drop-cap-view-default) .elementor-drop-cap{margin-top:8px}.elementor-widget-text-editor:not(.elementor-drop-cap-view-default) .elementor-drop-cap-letter{width:1em;height:1em}.elementor-widget-text-editor .elementor-drop-cap{float:left;text-align:center;line-height:1;font-size:50px}.elementor-widget-text-editor .elementor-drop-cap-letter{display:inline-block}<\/style>\t\t\t\t<p class=\"\">Le valoctocog\u00e8ne roxaparvovec est une th\u00e9rapie g\u00e9nique exp\u00e9rimentale (virus ad\u00e9noassoci\u00e9 [AAV] d\u00e9livrant le g\u00e8ne du facteur VIII d\u00e9l\u00e9t\u00e9 du domaine B) en cours de d\u00e9veloppement par BioMarin pour le traitement de l\u2019h\u00e9mophilie A. Cent trente-quatre patients ont re\u00e7u le traitement dans l\u2019\u00e9tude clinique de phase III de BioMarin. Dans l\u2019\u00e9tude clinique de phase I\/II en cours, dont le recrutement s\u2019est termin\u00e9 en 2017, 15 patients sont toujours suivis selon les recommandations des autorit\u00e9s r\u00e9glementaires.<\/p>\n<p class=\"\">\u00ab \u00c9v\u00e9nement ind\u00e9sirable grave \u00bb (EIG) est le terme utilis\u00e9 pour d\u00e9crire la survenue d\u2019un probl\u00e8me de sant\u00e9 grave au cours d\u2019une \u00e9tude, que le traitement exp\u00e9rimental en soit ou non \u00e0 l\u2019origine.<\/p>\n<p class=\"\">En f\u00e9vrier 2022, au cours du congr\u00e8s de l\u2019EAHAD, le docteur Johnny Mahlangu a fait \u00e9tat d\u2019un seul \u00e9v\u00e9nement ind\u00e9sirable grave chez l\u2019un des participants \u00e0 l\u2019\u00e9tude de phase I\/II ayant re\u00e7u la th\u00e9rapie g\u00e9nique il y a plus de cinq ans. En novembre 2021, une grosseur est apparue chez ce participant, au niveau du cou, diagnostiqu\u00e9e ult\u00e9rieurement comme cancer de la glande salivaire. La masse canc\u00e9reuse a \u00e9t\u00e9 retir\u00e9e et le patient plac\u00e9 sous surveillance. L\u2019investigateur de l\u2019\u00e9tude a jug\u00e9 l\u2019EIG comme non li\u00e9 au traitement et l\u2019a signal\u00e9 comme tel.<\/p>\n<p class=\"\">Un comit\u00e9 compos\u00e9 de l\u2019\u00e9quipe de l\u2019\u00e9tude de BioMarin, de l\u2019investigateur de l\u2019\u00e9tude, d\u2019un comit\u00e9 d\u2019experts ind\u00e9pendant qui proc\u00e8de \u00e0 la surveillance r\u00e9guli\u00e8re de l\u2019\u00e9tude (comit\u00e9 de surveillance des donn\u00e9es ou DMC) et d\u2019autres experts m\u00e9dicaux et scientifiques ont examin\u00e9 l\u2019EIG et l\u2019ont communiqu\u00e9 aux autorit\u00e9s r\u00e9glementaires. Au moment de l\u2019annonce, l\u2019EIG n\u2019\u00e9tait pas suspect\u00e9 comme \u00e9tant li\u00e9 au valoctocog\u00e8ne roxaparvovec. BioMarin a alors entrepris une analyse g\u00e9nomique \u00e0 partir d\u2019un \u00e9chantillon de tissu contenant la masse.<\/p>\n<p class=\"\">Fin avril, BioMarin a annonc\u00e9 les conclusions de cette analyse g\u00e9nomique : \u00ab les r\u00e9sultats ont montr\u00e9 un sch\u00e9ma d\u2019int\u00e9gration comparable entre tissus sains et tumoraux, sans qu\u2019\u00e9merge la preuve que l\u2019int\u00e9gration du vecteur ait contribu\u00e9 \u00e0 la masse de la glande salivaire \u00bb. Les donn\u00e9es seront pr\u00e9sent\u00e9es pour discussion le lundi 9 mai de 13 h 30 \u00e0 14 h 30 lors de la session sur les recherches cliniques r\u00e9centes du World Congress 2022 de la F\u00e9d\u00e9ration mondiale de l\u2019h\u00e9mophilie.<\/p>\n<p class=\"\">BioMarin a aussi communiqu\u00e9 ces donn\u00e9es \u00e0 l\u2019Agence europ\u00e9enne des m\u00e9dicaments (EMA) et \u00e0 la <em>Food and Drug Administration<\/em> (FDA) dans le cadre de la revue continue de la demande d\u2019autorisation de mise sur le march\u00e9 (AMM) d\u2019une part et du renouvellement de la demande de licence de produit biologique (<em>Biologics License Application<\/em>, BLA) d\u2019autre part.<\/p>\n<p class=\"\">Il s\u2019agit du troisi\u00e8me signalement de cancer chez les participants \u00e0 un essai de th\u00e9rapie g\u00e9nique dans l\u2019h\u00e9mophilie. Il a \u00e9t\u00e9 fait \u00e9tat d\u2019un cancer dans l\u2019essai FIX Hope-B (<a href=\"https:\/\/www.ehc.eu\/8702\" target=\"_blank\" rel=\"noopener\">D\u00e9claration de l\u2019EHC<\/a>, <a href=\"https:\/\/www.hemophilia.org\/news\/uniqure-announces-findings-from-hcc-case-investigation\" target=\"_blank\" rel=\"noopener\">D\u00e9claration de la NHF<\/a>, <a href=\"https:\/\/www.hemophilia.org\/news\/fda-puts-clinical-hold-on-uniqures-hope-b-trial\" target=\"_blank\" rel=\"noopener\">D\u00e9claration de la FMH<\/a>), chez un patient de sexe masculin pr\u00e9sentant plusieurs facteurs de risque associ\u00e9s au d\u00e9veloppement d\u2019un cancer h\u00e9patique. Ces facteurs comprenaient des ant\u00e9c\u00e9dents d\u2019h\u00e9patite C (trait\u00e9e avant le recrutement), l\u2019exposition au virus de l\u2019h\u00e9patite B, des preuves de st\u00e9atose h\u00e9patique non alcoolique et un \u00e2ge avanc\u00e9. Apr\u00e8s analyse d\u2019une biopsie pour l\u2019int\u00e9gration du syst\u00e8me de d\u00e9livrance du g\u00e8ne th\u00e9rapeutique (g\u00e9nome du vecteur AAV) dans les cellules, aucune diff\u00e9rence n\u2019a \u00e9t\u00e9 constat\u00e9e entre les tissus h\u00e9patiques sains et canc\u00e9reux. Un cancer des amygdales a aussi \u00e9t\u00e9 signal\u00e9 par la docteure Barbara Konkle dans un article sur l\u2019essai BAX-335 (AAV-facteur IX), publi\u00e9 dans la <a href=\"https:\/\/doi.org\/10.1182\/blood.2019004625\" target=\"_blank\" rel=\"noopener\">revue <em>blood<\/em><\/a>, o\u00f9 aucune int\u00e9gration du g\u00e9nome du vecteur AAV n\u2019a \u00e9t\u00e9 trouv\u00e9e dans les tissus du cancer des amygdales et il a \u00e9t\u00e9 consid\u00e9r\u00e9 que l\u2019\u00e9v\u00e9nement \u00e9tait sans lien avec la th\u00e9rapie g\u00e9nique.<\/p>\n<p class=\"\">Le risque de cancer est en g\u00e9n\u00e9ral au centre de l\u2019attention de toutes les th\u00e9rapies g\u00e9niques. La FMH, l\u2019EHC et la NHF, au nom de la communaut\u00e9 des patients, restent vigilants et insistent sur le passage au crible et l\u2019examen de chacun des EIG dans ces th\u00e9rapies, comme dans toutes. Cela contribue \u00e0 garantir la s\u00e9curit\u00e9 future de ces traitements qui peuvent changer la vie. Tout en faisant preuve de vigilance, nous devons \u00e9galement rester conscients du risque de base de cancer dans la population g\u00e9n\u00e9rale, y compris chez les personnes atteintes de troubles de la coagulation. Outre le risque d\u2019une forme de cancer dans la population g\u00e9n\u00e9rale, il existe par ailleurs un risque accru de cancer du foie, comme indiqu\u00e9 par <em>l\u2019European Haemophilia Safety Surveillance System<\/em> (EUHASS), chez les patients atteints d\u2019h\u00e9mophilie ayant des ant\u00e9c\u00e9dents de VIH et d\u2019h\u00e9patite C ou B. Nous devons continuer \u00e0 demander aux investigateurs, aux cliniciens, aux entreprises et aux autorit\u00e9s r\u00e9glementaires de poursuivre leurs efforts pour distinguer un EIG d\u2019un risque de base g\u00e9n\u00e9ral de la mani\u00e8re la plus opportune possible de sorte que les patients puissent prendre les d\u00e9cisions concernant les futurs traitements sur la base de l\u2019ensemble des preuves disponibles.<\/p>\n<p class=\"\">La surveillance des probl\u00e8mes de s\u00e9curit\u00e9 pour une technologie nouvelle comme la th\u00e9rapie g\u00e9nique est essentielle pour notre communaut\u00e9. \u00c0 cette fin, <a href=\"https:\/\/wfh.org\/fr\/collecte-de-donnees\/#gtr\" target=\"_blank\" rel=\"noopener\">le Registre de la th\u00e9rapie g\u00e9nique<\/a> est en cours de d\u00e9veloppement pour enregistrer la plupart ou l\u2019ensemble des patients recevant une th\u00e9rapie g\u00e9nique \u00e0 mesure que ces produits sont autoris\u00e9s.<\/p>\n<p class=\"\">Les trois organisations continueront \u00e0 surveiller et \u00e0 communiquer tous les \u00e9v\u00e9nements li\u00e9s \u00e0 la s\u00e9curit\u00e9 dans tous les domaines th\u00e9rapeutiques relevant de notre communaut\u00e9 de patients.<\/p>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-df88c2c elementor-widget elementor-widget-spacer\" data-id=\"df88c2c\" data-element_type=\"widget\" data-widget_type=\"spacer.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t<style>\/*! elementor - 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https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>BioMarin fait suite \u00e0 un \u00e9v\u00e9nement ind\u00e9sirable grave pr\u00e9c\u00e9demment signal\u00e9 dans son essai clinique de th\u00e9rapie g\u00e9nique de phase I\/II pour l\u2019h\u00e9mophilie A | FMH - F\u00e9d\u00e9ration mondiale de l\u2019h\u00e9mophilie<\/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:\/\/wfh.org\/fr\/article\/biomarin-fait-suite-a-un-evenement-indesirable-grave-precedemment-signale-dans-son-essai-clinique-de-therapie-genique-de-phase-i-ii-pour-lhemophilie-a\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"BioMarin fait suite \u00e0 un \u00e9v\u00e9nement ind\u00e9sirable grave pr\u00e9c\u00e9demment signal\u00e9 dans son essai clinique de th\u00e9rapie g\u00e9nique de phase I\/II pour l\u2019h\u00e9mophilie A\" \/>\n<meta property=\"og:description\" content=\"5 mai 2022 \u2013 Cette d\u00e9claration fait r\u00e9f\u00e9rence \u00e0 un \u00e9v\u00e9nement ind\u00e9sirable grave signal\u00e9 dans l\u2019essai clinique de th\u00e9rapie g\u00e9nique de phase I\/II avec le valoctocog\u00e8ne roxaparvovec pour l\u2019h\u00e9mophilie.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/wfh.org\/fr\/article\/biomarin-fait-suite-a-un-evenement-indesirable-grave-precedemment-signale-dans-son-essai-clinique-de-therapie-genique-de-phase-i-ii-pour-lhemophilie-a\/\" \/>\n<meta property=\"og:site_name\" content=\"FMH - 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