{"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\/pt\/three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe\/","title":{"rendered":"Tr\u00eas ensaios cl\u00ednicos em andamento da terapia gen\u00e9tica Elevidys foram temporariamente interrompidos na Europa"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Tr\u00eas ensaios cl\u00ednicos em andamento de ELEVIDYS (delandistrogene moxeparvovec-rokl), uma terapia gen\u00e9tica desenvolvida pela Sarepta e comercializada pela Roche, foram temporariamente interrompidos pela Ag\u00eancia Europeia de Medicamentos (EMA). Esta not\u00edcia esperada, mas n\u00e3o indesejada, causou profunda tristeza na comunidade da distrofia muscular de Duchenne (DMD).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Os ensaios cl\u00ednicos interrompidos incluem o estudo de fase 3 Envision, patrocinado pela Sarepta, que est\u00e1 avaliando Elevidys em meninos de 8 a 17 anos; o estudo de fase 2 Envol da Roche, que est\u00e1 investigando Elevidys em beb\u00eas e rec\u00e9m-nascidos; e o Estudo 104 da Sarepta, uma an\u00e1lise de fase 1 de Elevidys em meninos de 4 a 9 anos com anticorpos preexistentes para um sorotipo espec\u00edfico.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Antes de Roche, Sarepta e EMA, a primeira comunidade a publicar este an\u00fancio foi nosso parceiro turco &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 emitiu uma carta \u00e0 comunidade que voc\u00ea pode ler aqui.<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>\u00cdndice<\/h2><nav><ul><li><a href=\"#why-were-elevidys-clinical-trials-halted\">Por que os ensaios cl\u00ednicos do Elevidys foram interrompidos?<\/a><ul><li><a href=\"#srp-9001-104\">SRP-9001-104 Temporariamente Interrompido<\/a><\/li><li><a href=\"#envol-study-302\">ENVOL (Estudo 302) Interrompido Temporariamente<\/a><\/li><li><a href=\"#srp-9001-303\">SRP-9001-303 Temporariamente Interrompido<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-were-elevidys-clinical-trials-halted\">Por que os ensaios cl\u00ednicos do Elevidys foram interrompidos?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ap\u00f3s a morte de um garoto de 16 anos com distrofia muscular de Duchenne que recebeu uma infus\u00e3o de Elevidys em 18 de mar\u00e7o de 2025, a perspectiva para a terapia gen\u00e9tica, que \u00e9 comercializada a um pre\u00e7o comercial de $3 milh\u00f5es, mudou abruptamente.<a href=\"https:\/\/dmdwarrior.com\/pt\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\">Ler mais<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">At\u00e9 que a causa precisa da morte de um adolescente dos EUA que desenvolveu insufici\u00eancia hep\u00e1tica aguda (ALF) ap\u00f3s tomar Elevidys possa ser determinada, a Ag\u00eancia Europeia de Medicamentos (EMA) solicitou que os estudos fossem suspensos. Quando o paciente faleceu no m\u00eas passado, a Sarepta declarou que alteraria o r\u00f3tulo do Elevidys para incluir o sinal de seguran\u00e7a.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-104\">SRP-9001-104 Temporariamente Interrompido<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"113\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg\" alt=\"Ensaio cl\u00ednico SRP-9001-104 da terapia gen\u00e9tica Elevidys interrompido temporariamente na Europa\" 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=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Fonte: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512624-11-00?lang=en\" target=\"_blank\" rel=\"noopener\">Ensaio cl\u00ednico do SRP-9001-104<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"envol-study-302\">ENVOL (Estudo 302) Interrompido Temporariamente<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"272\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg\" alt=\"Ensaio cl\u00ednico ENVOL (Estudo 302) da terapia gen\u00e9tica Elevidys interrompido temporariamente na Europa\" 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=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Fonte: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2023-509901-57-00?lang=en\" target=\"_blank\" rel=\"noopener\">Ensaio cl\u00ednico do SRP-9001-302<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-303\">SRP-9001-303 Temporariamente Interrompido<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"768\" height=\"313\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg\" alt=\"Ensaio cl\u00ednico SRP-9001-303 da terapia gen\u00e9tica Elevidys temporariamente interrompido na Europa\" 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=\"auto, (max-width: 768px) 100vw, 768px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Fonte: <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512626-28-00?lang=en\" target=\"_blank\" rel=\"noopener\">Ensaio cl\u00ednico do SRP-9001-303<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Saber mais: <a href=\"https:\/\/dmdwarrior.com\/pt\/dmd-warriors-turkish-representative-shares-his-views-on-elevidys-gene-therapy-is-it-effective-and-why-is-it-expensive\/\">O representante turco do DMD WarrioR compartilha suas opini\u00f5es sobre a terapia gen\u00e9tica 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"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/3477","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=3477"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/3477\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/3478"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=3477"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=3477"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=3477"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}