{"id":3929,"date":"2025-06-17T16:48:33","date_gmt":"2025-06-17T13:48:33","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3929"},"modified":"2025-06-17T16:48:34","modified_gmt":"2025-06-17T13:48:34","slug":"capricor-therapeutics-announces-bmd-orphan-drug-designation-and-dmd-program-regulatory-development-for-deramiocel-cap-1002","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/capricor-therapeutics-announces-bmd-orphan-drug-designation-and-dmd-program-regulatory-development-for-deramiocel-cap-1002\/","title":{"rendered":"Capricor Therapeutics annonce la d\u00e9signation de m\u00e9dicament orphelin BMD et le d\u00e9veloppement r\u00e9glementaire du programme DMD pour Deramiocel (CAP-1002)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Capricor Therapeutics, soci\u00e9t\u00e9 de biotechnologie d\u00e9veloppant des th\u00e9rapies transformatrices \u00e0 base de cellules et d&#039;exosomes pour le traitement de maladies rares, a annonc\u00e9 aujourd&#039;hui que la Food and Drug Administration (FDA) am\u00e9ricaine avait accord\u00e9 la d\u00e9signation de m\u00e9dicament orphelin \u00e0 Deramiocel (CAP-1002), son principal candidat-m\u00e9dicament pour la th\u00e9rapie cellulaire, pour le traitement potentiel de la dystrophie musculaire de Becker (DMB). Cette d\u00e9signation renforce la position strat\u00e9gique de Capricor, qui d\u00e9veloppe une plateforme enti\u00e8rement int\u00e9gr\u00e9e ciblant les complications cardiaques et squelettiques de la dystrophie musculaire et accro\u00eet le potentiel commercial de son actif principal. <a href=\"https:\/\/dmdwarrior.com\/fr\/what-is-deramiocel\/\">Qu&#039;est-ce que Deramiocel ? Que fait CAP-1002 ?<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Capricor Therapeutics avance avec assurance<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>L&#039;US FDA accorde la d\u00e9signation de m\u00e9dicament orphelin \u00e0 Deramiocel pour le traitement de la dystrophie musculaire de Becker, \u00e9largissant ainsi le champ d&#039;action de Capricor dans les maladies neuromusculaires<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Le Capricor reste sur la bonne voie pour la date du 31 ao\u00fbt 2025, pr\u00e9vue par la Prescription Drug User Fee Act (PDUFA) pour le Deramiocel dans la dystrophie musculaire de Duchenne, suite \u00e0 l&#039;inspection pr\u00e9alable \u00e0 l&#039;homologation r\u00e9ussie du FDA<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">La dystrophie musculaire de Becker, comme la dystrophie musculaire de Duchenne (DMD), est une maladie neuromusculaire progressive li\u00e9e \u00e0 l&#039;X qui entra\u00eene une d\u00e9t\u00e9rioration significative des muscles squelettiques et cardiaques au fil du temps. Le Deramiocel est en cours de d\u00e9veloppement pour traiter les deux aspects de la pathologie de la maladie, y compris la cardiomyopathie, un contributeur principal \u00e0 la morbidit\u00e9 et \u00e0 la mortalit\u00e9 chez les patients atteints \u00e0 la fois de DMO et de DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>La demande de licence de produit biologique (BLA) de Capricor pour Deramiocel dans la DMD reste en cours d&#039;examen prioritaire, avec une date d&#039;action cible PDUFA du 31 ao\u00fbt 2025.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Apprendre encore plus<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/capricor-therapeutics-deramiocel-mid-cycle-review-meeting-with-fda\/\">Capricor Therapeutics annonce la fin de la r\u00e9union d&#039;\u00e9valuation<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel (CAP-1002), the company\u2019s lead cell therapy candidate, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation strengthens Capricor\u2019s strategic [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3931,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[361,90,86,87,89,85,117,362,88],"class_list":["post-3929","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-bmd","tag-cap-1002","tag-capricor","tag-capricor-therapeutics","tag-cardiomyopathy","tag-deramiocel","tag-dmd","tag-dmd-cardiomyopathy","tag-fda"],"subtitle":"La d\u00e9signation de m\u00e9dicament orphelin pour la dystrophie musculaire de Becker a \u00e9t\u00e9 annonc\u00e9e par Capricor Therapeutics, et des progr\u00e8s r\u00e9glementaires ont \u00e9t\u00e9 r\u00e9alis\u00e9s pour le programme de dystrophie musculaire de Duchenne selon la soci\u00e9t\u00e9.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3929","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=3929"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3929\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/3931"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=3929"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=3929"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=3929"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}