{"id":3477,"date":"2025-04-01T17:31:55","date_gmt":"2025-04-01T14:31:55","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3477"},"modified":"2025-04-04T10:01:49","modified_gmt":"2025-04-04T07:01:49","slug":"three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe\/","title":{"rendered":"Trois essais cliniques en cours sur la th\u00e9rapie g\u00e9nique Elevidys temporairement interrompus en Europe"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Trois essais cliniques en cours sur ELEVIDYS (delandistrogene moxeparvovec-rokl), une th\u00e9rapie g\u00e9nique d\u00e9velopp\u00e9e par Sarepta et commercialis\u00e9e par Roche, ont \u00e9t\u00e9 temporairement interrompus par l&#039;Agence europ\u00e9enne des m\u00e9dicaments (EMA). Cette nouvelle attendue, mais non d\u00e9plaisante, a suscit\u00e9 une profonde tristesse au sein de la communaut\u00e9 des personnes atteintes de dystrophie musculaire de Duchenne (DMD).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Les essais suspendus comprennent l&#039;\u00e9tude de phase 3 Envision sponsoris\u00e9e par Sarepta, qui \u00e9value le Elevidys chez les gar\u00e7ons \u00e2g\u00e9s de 8 \u00e0 17 ans ; l&#039;\u00e9tude de phase 2 Envol de Roche, qui \u00e9tudie le Elevidys chez les b\u00e9b\u00e9s et les nouveau-n\u00e9s ; et l&#039;\u00e9tude 104 de Sarepta, une \u00e9tude de phase 1 sur le Elevidys chez les gar\u00e7ons \u00e2g\u00e9s de 4 \u00e0 9 ans pr\u00e9sentant des anticorps pr\u00e9existants contre un s\u00e9rotype particulier.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avant Roche, Sarepta et EMA, la premi\u00e8re communaut\u00e9 \u00e0 publier cette annonce \u00e9tait notre partenaire turc &gt;&gt;&gt; <a href=\"https:\/\/dmd.arti.net\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD Dayanisma<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Roche a publi\u00e9 une lettre \u00e0 la communaut\u00e9 que vous pouvez lire ici.<a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/March-2025-Roche-DMD-Temporary-Clinical-Study-Halt-Community-Letter_31Mar25.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">PDF<\/a>)<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>Table des mati\u00e8res<\/h2><nav><ul><li><a href=\"#why-were-elevidys-clinical-trials-halted\">Pourquoi les essais cliniques sur le Elevidys ont-ils \u00e9t\u00e9 interrompus\u00a0?<\/a><ul><li><a href=\"#srp-9001-104\">SRP-9001-104 temporairement interrompu<\/a><\/li><li><a href=\"#envol-study-302\">ENVOL (\u00c9tude 302) temporairement interrompu<\/a><\/li><li><a href=\"#srp-9001-303\">SRP-9001-303 temporairement interrompu<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-were-elevidys-clinical-trials-halted\">Pourquoi les essais cliniques sur le Elevidys ont-ils \u00e9t\u00e9 interrompus\u00a0?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Apr\u00e8s le d\u00e9c\u00e8s d&#039;un gar\u00e7on de 16 ans atteint de dystrophie musculaire de Duchenne qui a re\u00e7u une perfusion de Elevidys le 18 mars 2025, les perspectives de la th\u00e9rapie g\u00e9nique, commercialis\u00e9e au prix commercial de $3 millions, ont chang\u00e9 brusquement.<a href=\"https:\/\/dmdwarrior.com\/fr\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\">En savoir plus<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">En attendant que la cause pr\u00e9cise du d\u00e9c\u00e8s d&#039;un adolescent am\u00e9ricain ayant d\u00e9velopp\u00e9 une insuffisance h\u00e9patique aigu\u00eb (IFA) apr\u00e8s la prise de Elevidys soit \u00e9tablie, l&#039;Agence europ\u00e9enne des m\u00e9dicaments (EMA) a demand\u00e9 la suspension des \u00e9tudes. Suite au d\u00e9c\u00e8s du patient le mois dernier, Sarepta a annonc\u00e9 qu&#039;elle modifierait l&#039;\u00e9tiquette du Elevidys afin d&#039;y inclure le signal de s\u00e9curit\u00e9.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-104\">SRP-9001-104 temporairement interrompu<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img decoding=\"async\" width=\"762\" height=\"113\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg\" alt=\"Essai clinique SRP-9001-104 de th\u00e9rapie g\u00e9nique Elevidys temporairement interrompu en Europe\" class=\"wp-image-3480\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-300x44.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-18x3.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-150x22.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-696x103.jpg 696w\" sizes=\"(max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Source: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512624-11-00?lang=en\" target=\"_blank\" rel=\"noopener\">Essai clinique du SRP-9001-104<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"envol-study-302\">ENVOL (\u00c9tude 302) temporairement interrompu<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img decoding=\"async\" width=\"762\" height=\"272\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg\" alt=\"Essai clinique ENVOL (\u00e9tude 302) sur la th\u00e9rapie g\u00e9nique Elevidys temporairement interrompu en Europe\" class=\"wp-image-3481\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-300x107.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-18x6.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-150x54.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-696x248.jpg 696w\" sizes=\"(max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Source: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2023-509901-57-00?lang=en\" target=\"_blank\" rel=\"noopener\">Essai clinique du SRP-9001-302<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-303\">SRP-9001-303 temporairement interrompu<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img decoding=\"async\" width=\"768\" height=\"313\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg\" alt=\"Essai clinique SRP-9001-303 de th\u00e9rapie g\u00e9nique Elevidys temporairement interrompu en Europe\" class=\"wp-image-3482\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-300x122.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-18x7.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-150x61.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-696x284.jpg 696w\" sizes=\"(max-width: 768px) 100vw, 768px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Source: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512626-28-00?lang=en\" target=\"_blank\" rel=\"noopener\">Essai clinique du SRP-9001-303<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Apprendre encore plus: <a href=\"https:\/\/dmdwarrior.com\/fr\/dmd-warriors-turkish-representative-shares-his-views-on-elevidys-gene-therapy-is-it-effective-and-why-is-it-expensive\/\">Le repr\u00e9sentant turc de DMD WarrioR partage son point de vue sur la th\u00e9rapie g\u00e9nique Elevidys.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the European Medicines Agency (EMA). This expected but not unwelcome news has caused deep sadness in the Duchenne muscular dystrophy (DMD) community. The paused trials include the Sarepta-sponsored phase 3 Envision study, [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3478,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,33,179,91,84,213,61,203,65],"class_list":["post-3477","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-elevidys","tag-elevidys-gene-therapy","tag-ema","tag-europe","tag-european-medicines-agency","tag-gene-therapy","tag-roche","tag-sarepta"],"subtitle":"Suite au d\u00e9c\u00e8s d'un enfant de 16 ans recevant une perfusion de Elevidys, 3 essais cliniques en cours en Europe (\u00e9tude 104, \u00e9tude 302 et \u00e9tude 303) ont \u00e9t\u00e9 temporairement interrompus.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3477","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=3477"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/3477\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/3478"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=3477"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=3477"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=3477"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}