{"id":4826,"date":"2025-10-21T14:58:09","date_gmt":"2025-10-21T11:58:09","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4826"},"modified":"2025-11-01T16:06:07","modified_gmt":"2025-11-01T13:06:07","slug":"why-elevidys-was-not-approved-by-the-european-medicines-agency-ema","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/","title":{"rendered":"Elevidys Neden Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) Taraf\u0131ndan Onaylanmad\u0131?"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Elevidys (etkin madde: delandistrogene moxeparvovec) gen terapisi, bu hasta grubunda kan\u0131tlanm\u0131\u015f faydas\u0131na ili\u015fkin \u00f6nemli endi\u015feler nedeniyle, Duchenne kas distrofisi (DMD) olan 3-7 ya\u015f aras\u0131 ayakta tedavi g\u00f6ren \u00e7ocuklar\u0131n tedavisi i\u00e7in Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) taraf\u0131ndan onaylanmam\u0131\u015ft\u0131r.<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noopener\">Avrupa \u0130la\u00e7 Ajans\u0131 (EMA)<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u0130\u015fte ba\u015fl\u0131ca sebepler:<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#reasons-for-elevidys-refusal-by-the-ema\">Elevidys&#039;nin EMA Taraf\u0131ndan Reddedilmesinin Nedenleri<\/a><ul><li><a href=\"#additional-context-and-concerns\">Ek ba\u011flam ve endi\u015feler<\/a><\/li><li><a href=\"#summary\">\u00d6zet<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"reasons-for-elevidys-refusal-by-the-ema\">Elevidys&#039;nin EMA Taraf\u0131ndan Reddedilmesinin Nedenleri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>EMA&#039;nin kamuoyu de\u011ferlendirmesine g\u00f6re belirli nedenler \u015funlard\u0131r:<\/strong><\/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.<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">Avrupa \u0130la\u00e7 Ajans\u0131 (EMA)<\/a>) <strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">North Star Ambulatuvar De\u011ferlendirmesi Nedir?<\/a><\/li>\n\n\n\n<li>Terapi distrofin proteininin daha k\u0131sa bir formunun ifadesini ind\u00fcklese de, bu distrofin ifadesinin d\u00fczeyleri hareket yetene\u011finde anlaml\u0131 iyile\u015ftirmelerle g\u00fcvenilir bir \u015fekilde ili\u015fkilendirilemedi.<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">Avrupa \u0130la\u00e7 Ajans\u0131 (EMA)<\/a>)<\/li>\n\n\n\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.<a href=\"https:\/\/www.institut-myologie.org\/en\/2025\/09\/22\/unfavorable-opinion-on-marketing-authorisation-and-suspension-of-clinical-trials-in-europe-for-elevidys-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">institut-myologie.org<\/a>)<\/li>\n\n\n\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.<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">Avrupa \u0130la\u00e7 Ajans\u0131 (EMA)<\/a>)<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"additional-context-and-concerns\">Ek ba\u011flam ve endi\u015feler<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>G\u00fcvenlik sorunlar\u0131: \u00d6zellikle y\u00fcr\u00fcyemeyen hastalarda \u00f6nemli g\u00fcvenlik endi\u015feleri de vard\u0131. Elevidys alan y\u00fcr\u00fcyemeyen hastalarda akut karaci\u011fer yetmezli\u011finden kaynaklanan birka\u00e7 \u00f6l\u00fcm bildirildi, bu pop\u00fclasyondaki \u00e7al\u0131\u015fmalar durduruldu.<a href=\"https:\/\/dmdwarrior.com\/tr\/aan-report-reviews-the-state-of-evidence-on-elevidys-gene-therapy-cure-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD Sava\u015f\u00e7\u0131s\u0131<\/a>) EMA&#039;nin reddetmesi \u00f6ncelikle ayakta tedavi g\u00f6ren 3-7 ya\u015f aras\u0131 pop\u00fclasyon i\u00e7in etkililik gerek\u00e7esine dayansa da, g\u00fcvenlik sinyali muhtemelen genel d\u00fczenleyici ortama da yans\u0131m\u0131\u015ft\u0131r.<\/li>\n\n\n\n<li>EMA&#039;ye sunulan ba\u015fvuru, 3-7 ya\u015f aras\u0131 ayakta tedavi g\u00f6ren hastalar i\u00e7indi. EMA, \u00e7al\u0131\u015fman\u0131n tasar\u0131m\u0131n\u0131n ve birincil sonlan\u0131m noktas\u0131n\u0131n (NSAA&#039;da 12 ayl\u0131k de\u011fi\u015fim) se\u00e7iminin, bu grupta hastal\u0131k ilerlemesinde anlaml\u0131 bir yava\u015flama oldu\u011funu g\u00f6stermek i\u00e7in yetersiz oldu\u011funa karar verdi.<a href=\"https:\/\/www.institut-myologie.org\/en\/2025\/09\/22\/unfavorable-opinion-on-marketing-authorisation-and-suspension-of-clinical-trials-in-europe-for-elevidys-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">institut-myologie.org<\/a>)<\/li>\n\n\n\n<li>Terapi, farkl\u0131 d\u00fczenleyici \u00e7er\u00e7eveler alt\u0131nda baz\u0131 di\u011fer b\u00f6lgelerde (\u00f6rne\u011fin Japonya ve baz\u0131 Orta Do\u011fu \u00fclkeleri) onaylanm\u0131\u015ft\u0131.<a href=\"https:\/\/dmdwarrior.com\/tr\/in-which-countries-is-elevidys-approved-is-elevidys-approved-\/\" target=\"_blank\" rel=\"noreferrer noopener\">dmdwarrior.com<\/a>)<\/li>\n\n\n\n<li>EMA&#039;nin g\u00f6r\u00fc\u015f\u00fc (\u0130nsan Kullan\u0131m\u0131na Y\u00f6nelik T\u0131bbi \u00dcr\u00fcnler Komitesi, CHMP arac\u0131l\u0131\u011f\u0131yla) Avrupa Komisyonu karar\u0131na kadar hukuken ba\u011flay\u0131c\u0131 de\u011fildir, ancak pratikte CHMP&#039;nin olumsuz g\u00f6r\u00fc\u015fleri neredeyse her zaman ret ile sonu\u00e7lanmaktad\u0131r.<a href=\"https:\/\/www.globenewswire.com\/de\/news-release\/2025\/08\/11\/3131260\/32716\/en\/sarepta-therapeutics-srpt-declines-again-on-ema-recommendation-to-refuse-elevidys-marketing-authorization-securities-class-action-pending-hagens-berman.html\" target=\"_blank\" rel=\"noreferrer noopener\">GlobeNewswire<\/a>)<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"summary\">\u00d6zet<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys, DMD i\u00e7in yeni bir gen tedavisi yakla\u015f\u0131m\u0131 sunmas\u0131na ra\u011fmen, EMA sunulan kan\u0131tlar\u0131n hedeflenen hareketli \u00e7ocuklarda (3-7 ya\u015f) motor fonksiyonu iyile\u015ftirdi\u011fini yeterince g\u00f6stermedi\u011fi ve bu pop\u00fclasyon i\u00e7in fayda-risk oran\u0131n\u0131n belirlenmedi\u011fi sonucuna vard\u0131. Bu nedenle pazarlama yetkisinin reddedilmesini \u00f6nerdiler.<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/elevidys\" target=\"_blank\" rel=\"noreferrer noopener\">Avrupa \u0130la\u00e7 Ajans\u0131 (EMA)<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Devam\u0131n\u0131 oku<\/strong>: <a href=\"https:\/\/www.ema.europa.eu\/en\/documents\/assessment-report\/elevidys-epar-refusal-public-assessment-report_en.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">EMA taraf\u0131ndan yay\u0131nlanan Elevidys reddinin gerek\u00e7elerine ili\u015fkin kapsaml\u0131 rapora bu ba\u011flant\u0131dan ula\u015fabilirsiniz.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>The Elevidys (active substance: delandistrogene\u202fmoxeparvovec) gene therapy was not approved by the European Medicines Agency (EMA) for the treatment of ambulatory children aged 3\u20117 years with Duchenne\u202fmuscular\u202fdystrophy (DMD) because of significant concerns about its demonstrated benefit in that patient population. (European Medicines Agency (EMA)) Here are the key reasons: Reasons for Elevidys&#8217; Refusal by the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4833,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[121,33,61,65],"class_list":["post-4826","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-gene-therapy","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4826","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=4826"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4826\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4833"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4826"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4826"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4826"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}