{"id":4222,"date":"2025-07-25T14:46:11","date_gmt":"2025-07-25T11:46:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4222"},"modified":"2025-10-21T14:29:47","modified_gmt":"2025-10-21T11:29:47","slug":"the-european-medicines-agency-ema-issued-a-negative-opinion-on-elevidys","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/the-european-medicines-agency-ema-issued-a-negative-opinion-on-elevidys\/","title":{"rendered":"L&#039;Agence europ\u00e9enne des m\u00e9dicaments (EMA) a \u00e9mis un avis n\u00e9gatif sur Elevidys"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Roche a annonc\u00e9 aujourd&#039;hui que le Comit\u00e9 des m\u00e9dicaments \u00e0 usage humain (CHMP) de l&#039;Agence europ\u00e9enne des m\u00e9dicaments (EMA) a \u00e9mis un avis n\u00e9gatif sur l&#039;autorisation de mise sur le march\u00e9 conditionnelle (AMM) du Elevidys (delandistrogene moxeparvovec) pour les personnes ambulatoires \u00e2g\u00e9es de trois \u00e0 sept ans atteintes de dystrophie musculaire de Duchenne (DMD). <a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">En savoir plus<\/a> \u2013 <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Compte tenu du besoin important non satisfait en mati\u00e8re de DMD, Roche pr\u00e9voit de continuer \u00e0 travailler avec le EMA pour explorer une voie potentielle \u00e0 suivre.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Pourquoi le Elevidys n&#039;a pas \u00e9t\u00e9 approuv\u00e9 par l&#039;Agence europ\u00e9enne des m\u00e9dicaments (EMA)<\/strong><\/h2>\n\n\n\n<h4 class=\"wp-block-heading\">Principales raisons de refus<\/h4>\n\n\n\n<p class=\"wp-block-paragraph\">Selon l&#039;\u00e9valuation publique du EMA, les raisons sp\u00e9cifiques incluent :<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Dans l&#039;\u00e9tude pivot (principale preuve pr\u00e9sent\u00e9e), aucune am\u00e9lioration statistiquement significative de la fonction motrice (mesur\u00e9e selon l&#039;\u00e9chelle North Star Ambulatory Assessment, NSAA) n&#039;a \u00e9t\u00e9 observ\u00e9e \u00e0 12 mois entre le groupe Elevidys et le groupe placebo. La diff\u00e9rence n&#039;\u00e9tait que de 0,65 point sur une \u00e9chelle de 34 points, ce qui, selon l&#039;\u00e9tude EMA, \u201c pourrait \u00eatre d\u00fb au hasard \u201d.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Bien que la th\u00e9rapie ait induit l\u2019expression d\u2019une forme plus courte de la prot\u00e9ine dystrophine, les niveaux d\u2019expression de cette dystrophine n\u2019ont pas pu \u00eatre li\u00e9s de mani\u00e8re fiable \u00e0 des am\u00e9liorations significatives de la capacit\u00e9 de mouvement.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>M\u00eame en consid\u00e9rant des sous-groupes de patients qui semblaient mieux r\u00e9pondre, la soci\u00e9t\u00e9 n\u2019a pas pu d\u00e9montrer de mani\u00e8re convaincante que le traitement procurait un b\u00e9n\u00e9fice cliniquement significatif dans ces groupes non plus.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c9tant donn\u00e9 que la soci\u00e9t\u00e9 avait d\u00e9pos\u00e9 une demande d&#039;autorisation de mise sur le march\u00e9 conditionnelle (AMM), la norme EMA exige un rapport b\u00e9n\u00e9fice-risque positif (c&#039;est-\u00e0-dire une preuve de b\u00e9n\u00e9fice + un risque g\u00e9rable) appuy\u00e9 par des donn\u00e9es convaincantes. L&#039;AMM EMA a conclu que les b\u00e9n\u00e9fices n&#039;avaient pas \u00e9t\u00e9 suffisamment d\u00e9montr\u00e9s dans ce contexte et a donc refus\u00e9 l&#039;autorisation.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Apprendre encore plus:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/fr\/roche-suspends-shipments-of-elevidys-gene-therapy-outside-of-the-united-states\/\" target=\"_blank\" rel=\"noreferrer noopener\">Roche suspend les livraisons de th\u00e9rapie g\u00e9nique Elevidys hors des \u00c9tats-Unis<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD). &#8211; Read More &#8211; Given the high unmet need [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4226,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[121,33,179,91,213],"class_list":["post-4222","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-elevidys-gene-therapy","tag-ema","tag-european-medicines-agency"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4222","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=4222"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4222\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/4226"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=4222"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=4222"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=4222"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}