{"id":4222,"date":"2025-07-25T14:46:11","date_gmt":"2025-07-25T11:46:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4222"},"modified":"2025-10-21T14:29:47","modified_gmt":"2025-10-21T11:29:47","slug":"the-european-medicines-agency-ema-issued-a-negative-opinion-on-elevidys","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/the-european-medicines-agency-ema-issued-a-negative-opinion-on-elevidys\/","title":{"rendered":"Avrupa \u0130la\u00e7 Ajans\u0131 (EMA), Elevidys Hakk\u0131nda Olumsuz G\u00f6r\u00fc\u015f Bildirdi"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Roche, bug\u00fcn Avrupa \u0130la\u00e7 Ajans\u0131&#039;n\u0131n (EMA) \u0130nsan Kullan\u0131m\u0131na Y\u00f6nelik T\u0131bbi \u00dcr\u00fcnler Komitesi&#039;nin (CHMP), Duchenne kas distrofisi (DMD) olan \u00fc\u00e7 ila yedi ya\u015f aras\u0131 ayakta tedavi g\u00f6ren bireyler i\u00e7in Elevidys (delandistrogene moxeparvovec) i\u00e7in ko\u015fullu pazarlama yetkisi (CMA) hakk\u0131nda olumsuz g\u00f6r\u00fc\u015f bildirdi\u011fini duyurdu. <a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">Devam\u0131n\u0131 oku<\/a> \u2013 <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">DMD&#039;deki kar\u015f\u0131lanmam\u0131\u015f y\u00fcksek ihtiya\u00e7 g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda, Roche ileriye d\u00f6n\u00fck potansiyel bir yol ke\u015ffetmek i\u00e7in EMA ile \u00e7al\u0131\u015fmaya devam etmeyi planl\u0131yor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\"><strong>Elevidys Neden Avrupa \u0130la\u00e7 Ajans\u0131 Taraf\u0131ndan Onaylanmad\u0131 (EMA)<\/strong><\/h2>\n\n\n\n<h4 class=\"wp-block-heading\">Reddetmenin temel nedenleri<\/h4>\n\n\n\n<p class=\"wp-block-paragraph\">EMA&#039;nin kamuoyu de\u011ferlendirmesine g\u00f6re belirli nedenler \u015funlard\u0131r:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Temel \u00e7al\u0131\u015fmada (sunulan ana kan\u0131t), 12 ayda Elevidys kolu ile plasebo kolu aras\u0131nda motor fonksiyonda (North Star Ambulatuvar De\u011ferlendirmesi, NSAA kullan\u0131larak \u00f6l\u00e7\u00fcld\u00fc\u011f\u00fc \u00fczere) istatistiksel olarak anlaml\u0131 bir iyile\u015fme g\u00f6r\u00fclmedi. 34 puanl\u0131k bir \u00f6l\u00e7ekte fark sadece 0,65 puand\u0131 ve EMA bunun &quot;\u015fansa ba\u011fl\u0131 olabilece\u011fi&quot; sonucuna vard\u0131.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Terapi distrofin proteininin daha k\u0131sa bir formunun ekspresyonunu ind\u00fcklese de, bu distrofin ekspresyonunun seviyeleri hareket yetene\u011finde anlaml\u0131 iyile\u015fmelerle g\u00fcvenilir bir \u015fekilde ili\u015fkilendirilemedi.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Daha iyi yan\u0131t veren hasta alt gruplar\u0131 dikkate al\u0131nd\u0131\u011f\u0131nda bile \u015firket, tedavinin bu gruplarda klinik a\u00e7\u0131dan anlaml\u0131 bir fayda sa\u011flad\u0131\u011f\u0131n\u0131 ikna edici bir \u015fekilde g\u00f6steremedi.<\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u015eirket ko\u015fullu pazarlama izni (CMA) ba\u015fvurusunda bulundu\u011fundan, EMA&#039;nin uygulad\u0131\u011f\u0131 standart, olumlu bir fayda-risk (yani fayda kan\u0131t\u0131 + y\u00f6netilebilir risk) ve bunu destekleyecek ikna edici veriler olmas\u0131 gerekti\u011fidir. EMA, faydalar\u0131n bu ba\u011flamda yeterince kan\u0131tlanmad\u0131\u011f\u0131 sonucuna varm\u0131\u015f ve bu nedenle izni reddetmi\u015ftir.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/tr\/roche-suspends-shipments-of-elevidys-gene-therapy-outside-of-the-united-states\/\" target=\"_blank\" rel=\"noreferrer noopener\">Roche, Elevidys Gen Terapisinin ABD D\u0131\u015f\u0131na Sevkiyat\u0131n\u0131 Durdurdu<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD). &#8211; Read More &#8211; Given the high unmet need [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4226,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[121,33,179,91,213],"class_list":["post-4222","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-elevidys-gene-therapy","tag-ema","tag-european-medicines-agency"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4222","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=4222"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4222\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4226"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4222"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4222"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4222"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}