{"id":3996,"date":"2025-06-24T16:56:02","date_gmt":"2025-06-24T13:56:02","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3996"},"modified":"2025-06-24T16:56:03","modified_gmt":"2025-06-24T13:56:03","slug":"capricor-therapeutics-provides-regulatory-update-on-deramiocel-bla-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/capricor-therapeutics-provides-regulatory-update-on-deramiocel-bla-for-duchenne-muscular-dystrophy\/","title":{"rendered":"Capricor Therapeutics fournit une mise \u00e0 jour r\u00e9glementaire sur la BLA Deramiocel pour la dystrophie musculaire de Duchenne"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dans le cadre de l&#039;examen en cours du FDA, la Soci\u00e9t\u00e9 a \u00e9t\u00e9 inform\u00e9e qu&#039;aucune r\u00e9union du comit\u00e9 consultatif n&#039;est pr\u00e9vue pour le moment. La BLA reste en examen prioritaire, avec une date d&#039;action cible fix\u00e9e au 31 ao\u00fbt 2025 en vertu de la Loi sur les frais d&#039;utilisation des m\u00e9dicaments sur ordonnance (PDUFA).<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>FDA a indiqu\u00e9 qu&#039;une r\u00e9union du comit\u00e9 consultatif n&#039;est pas n\u00e9cessaire pour le moment.<\/li>\n\n\n\n<li>R\u00e9union d\u2019examen en personne de fin de cycle pr\u00e9vue \u00e0 la mi-juillet.<\/li>\n\n\n\n<li>La demande de licence de produits biologiques (BLA) reste sous examen prioritaire avec une date d&#039;action cible PDUFA fix\u00e9e au 31 ao\u00fbt 2025.<\/li>\n\n\n\n<li>Les donn\u00e9es sur quatre ans pr\u00e9sent\u00e9es lors de la conf\u00e9rence PPMD 2025 d\u00e9montrent une fonction cardiaque soutenue.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Capricor a r\u00e9cemment pr\u00e9sent\u00e9 les donn\u00e9es de quatre ans de son \u00e9tude d&#039;extension ouverte (OLE) HOPE-2 lors de la conf\u00e9rence Parent Project Muscular Dystrophy (PPMD) de 2025. Ces donn\u00e9es repr\u00e9sentent l&#039;un des plus anciens ensembles de donn\u00e9es th\u00e9rapeutiques sur la DMD, incluant des mesures de la fonction cardiaque et musculaire squelettique. Ces donn\u00e9es ont d\u00e9montr\u00e9 une pr\u00e9servation continue de la fonction cardiaque, ainsi qu&#039;un b\u00e9n\u00e9fice clinique durable du traitement \u00e0 long terme par Deramiocel.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En savoir plus: <a href=\"https:\/\/dmdwarrior.com\/fr\/capricor-therapeutics-announces-positive-4-year-data-of-deramiocel-cap-1002-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Capricor Therapeutics annonce des donn\u00e9es positives sur 4 ans pour Deramiocel (CAP-1002) dans la dystrophie musculaire de Duchenne<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Pourquoi les actions Capricor chutent-elles ?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">L&#039;action Capricor Therapeutics a chut\u00e9 lundi. L&#039;indice FDA a indiqu\u00e9 qu&#039;une r\u00e9union du comit\u00e9 consultatif n&#039;\u00e9tait pas actuellement requise pour la th\u00e9rapie cellulaire de la soci\u00e9t\u00e9 contre la dystrophie musculaire de Duchenne.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En juin, l&#039;entreprise a annonc\u00e9 que l&#039;inspection de son usine de fabrication du FDA n&#039;avait r\u00e9v\u00e9l\u00e9 aucun probl\u00e8me majeur. Cependant, des d\u00e9veloppements r\u00e9cents ont soulev\u00e9 des doutes quant \u00e0 l&#039;efficacit\u00e9 du traitement.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Les actions Capricor ont chut\u00e9 d&#039;environ 30% depuis vendredi suite \u00e0 la nouvelle.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Une organisation de d\u00e9fense des droits qui travaille dans le domaine des th\u00e9rapies cellulaires et g\u00e9niques a soulign\u00e9 l\u2019importance des d\u00e9cisions r\u00e9glementaires et des essais cliniques dans ce domaine.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Les actions Capricor se n\u00e9gocient \u00e0 $7,30, en baisse de 11,6% lundi.<\/p>","protected":false},"excerpt":{"rendered":"<p>As part of the FDA\u2019s ongoing review, the Company has been informed that an Advisory Committee meeting is not indicated at this time. The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3998,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[90,86,87,89,85,362,301],"class_list":["post-3996","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-cap-1002","tag-capricor","tag-capricor-therapeutics","tag-cardiomyopathy","tag-deramiocel","tag-dmd-cardiomyopathy","tag-hope-2"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3996","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=3996"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3996\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/3998"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=3996"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=3996"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=3996"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}