{"id":4357,"date":"2025-08-20T17:19:30","date_gmt":"2025-08-20T14:19:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4357"},"modified":"2025-08-20T17:21:43","modified_gmt":"2025-08-20T14:21:43","slug":"keros-announces-u-s-fda-orphan-drug-designation-granted-to-ker-065-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/keros-announces-u-s-fda-orphan-drug-designation-granted-to-ker-065-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Keros annonce la d\u00e9signation de m\u00e9dicament orphelin aux \u00c9tats-Unis FDA accord\u00e9e \u00e0 KER-065 pour le traitement de la dystrophie musculaire de Duchenne"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Keros Therapeutics a annonc\u00e9 aujourd&#039;hui que la Food and Drug Administration am\u00e9ricaine (\u00ab FDA \u00bb) a accord\u00e9 la d\u00e9signation de m\u00e9dicament orphelin \u00e0 KER-065 pour le traitement de la dystrophie musculaire de Duchenne (\u00ab DMD \u00bb).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00ab L&#039;obtention de la d\u00e9signation de m\u00e9dicament orphelin pour KER-065 souligne l&#039;important besoin m\u00e9dical non satisfait des patients atteints de DMD \u00bb, a d\u00e9clar\u00e9 Jasbir S. Seehra, pr\u00e9sident-directeur g\u00e9n\u00e9ral. \u00ab Cette d\u00e9signation constitue une \u00e9tape importante pour Keros, alors que nous avan\u00e7ons vers un essai clinique de phase 2 pour KER-065 chez des patients atteints de DMD. \u00bb<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Le FDA accorde la d\u00e9signation de m\u00e9dicament orphelin aux th\u00e9rapies exp\u00e9rimentales ciblant des maladies ou affections rares touchant moins de 200\u00a0000 personnes aux \u00c9tats-Unis. Cette d\u00e9signation offre certains avantages potentiels, notamment des cr\u00e9dits d&#039;imp\u00f4t pour les essais cliniques qualifi\u00e9s, une exon\u00e9ration ou un paiement partiel des frais de dossier FDA et sept ans d&#039;exclusivit\u00e9 commerciale, en cas d&#039;approbation.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/keros-therapeutics-announces-initial-topline-results-from-the-phase-1-clinical-trial-of-ker-065-in-healthy-volunteers\/\" target=\"_blank\" rel=\"noreferrer noopener\">Essai clinique de phase 1 du KER-065 chez des volontaires sains<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Qu&#039;est-ce que KER-065 ?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">KER-065 est un nouveau pi\u00e8ge \u00e0 ligand compos\u00e9 d&#039;un domaine de liaison au ligand modifi\u00e9 d\u00e9riv\u00e9 du r\u00e9cepteur d&#039;activine de type IIA et du r\u00e9cepteur d&#039;activine de type IIB qui est fusionn\u00e9 \u00e0 la partie de l&#039;anticorps humain connue sous le nom de domaine Fc. KER-065 est con\u00e7u pour agir comme un pi\u00e8ge \u00e0 ligand et inhiber les effets biologiques de la myostatine et de l&#039;activine A, deux ligands qui signalent via les r\u00e9cepteurs d&#039;activine, pour augmenter la r\u00e9g\u00e9n\u00e9ration du muscle squelettique, augmenter la taille et la force musculaire, r\u00e9duire la graisse corporelle, r\u00e9duire la fibrose du muscle squelettique et augmenter la r\u00e9sistance osseuse.<\/p>","protected":false},"excerpt":{"rendered":"<p>Keros Therapeutics today announced the U.S. Food and Drug Administration (\u201cFDA\u201d) granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (\u201cDMD\u201d). \u201cReceiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD,\u201d said Jasbir S. Seehra, President and Chief Executive Officer. \u201cThis designation serves as a [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4360,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[337,336,195],"class_list":["post-4357","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ker-065","tag-keros-therapeutics","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4357","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=4357"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/4357\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/4360"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=4357"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=4357"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=4357"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}