{"id":5141,"date":"2025-11-13T11:00:25","date_gmt":"2025-11-13T08:00:25","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5141"},"modified":"2025-11-13T11:10:04","modified_gmt":"2025-11-13T08:10:04","slug":"why-does-the-fda-approve-dmd-therapies-that-fail-in-clinical-trials","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/why-does-the-fda-approve-dmd-therapies-that-fail-in-clinical-trials\/","title":{"rendered":"FDA Klinik \u00c7al\u0131\u015fmalarda Ba\u015far\u0131s\u0131z Olan DMD Terapilerini Neden Onayl\u0131yor?"},"content":{"rendered":"

Milyonlarca dolara pazarlanan gen terapileri ve her ay uygulan\u0131p binlerce dolara sat\u0131lan ekzon atlama terapileri ger\u00e7ekten i\u015fe yar\u0131yor mu?<\/strong> Terapilere eri\u015fim i\u00e7in yo\u011fun stres alt\u0131nda kampanya y\u00fcr\u00fcten aileler, DMD terapilerinin \u00e7ocuklar\u0131n\u0131n hayatlar\u0131n\u0131 olumlu y\u00f6nde etkileyece\u011fini umuyor ancak veriler pek de umut verici de\u011fil.<\/p>\n\n\n\n

Sarepta Therapeutics'nin bu ay, Duchenne kas distrofisi i\u00e7in pazarlanan iki ekzon atlama ilac\u0131na y\u00f6nelik do\u011frulay\u0131c\u0131 bir denemenin birincil sonlan\u0131m noktas\u0131na ula\u015fmad\u0131\u011f\u0131n\u0131 duyurmas\u0131, ila\u00e7lar\u0131n sonu anlam\u0131na gelebilirdi. >> Sarepta'n\u0131n DMD Ekzon Atlama Terapileri Do\u011frulay\u0131c\u0131 \u00c7al\u0131\u015fmada Ba\u015far\u0131s\u0131z Oldu, Hisse Senedi Fiyat\u0131 D\u00fc\u015ft\u00fc<\/a><\/p>\n\n\n\n

Ancak \u015firket liderleri bu heyecan\u0131 kaybetmeye yana\u015fmad\u0131. Ya\u015flar\u0131 6 ile 13 aras\u0131nda de\u011fi\u015fen ara\u015ft\u0131rma kat\u0131l\u0131mc\u0131lar\u0131nda g\u00f6zlemlenen klinik a\u00e7\u0131dan \u00f6nemli ek de\u011fi\u015fikliklere dayanarak, FDA ile "h\u0131zland\u0131r\u0131lm\u0131\u015f onaydan geleneksel onaya ge\u00e7i\u015f" hakk\u0131nda g\u00f6r\u00fc\u015fmek \u00fczere bir araya gelmeyi planl\u0131yorlar.\u201c<\/p>\n\n\n\n

Ba\u015far\u0131s\u0131z bir deneme her zaman \u00f6l\u00fcm cezas\u0131 anlam\u0131na gelmez ve Sarepta bunu fark eden ilk \u015firket de\u011fil; hatta bir ilac\u0131 kesin \u00f6l\u00fcmden kurtard\u0131. Son a\u015famadaki bir deneme birincil son noktaya ula\u015famamas\u0131na ra\u011fmen, DMD gen tedavisi olan Elevidys tamamen onayland\u0131 ve etiket geni\u015fletmesi yap\u0131ld\u0131. >> Elevidys Neden Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) Taraf\u0131ndan Onaylanmad\u0131?<\/a><\/p>\n\n\n\n

Bu uyarlanabilir stratejinin muhalifleri de var. Baz\u0131 uzmanlara g\u00f6re, bir ila\u00e7 denemesi ba\u015far\u0131s\u0131z oldu\u011funda \u015firket kesin bir durdurma karar\u0131 almal\u0131.<\/p>\n\n\n\n

\u201cYale T\u0131p Fak\u00fcltesi'nde t\u0131p ve halk sa\u011fl\u0131\u011f\u0131 profes\u00f6r\u00fc olan Dr. Joseph Ross, "Akl\u0131mda hi\u00e7bir fikir birli\u011fi yok," dedi. "Bir ilac\u0131n i\u015fe yaray\u0131p yaramad\u0131\u011f\u0131n\u0131 g\u00f6stermek i\u00e7in tasarlanm\u0131\u015f bir deneyin, e\u011fer ba\u015far\u0131s\u0131z olursa, nas\u0131l olup da kullan\u0131m onay\u0131 alabildi\u011fini anlayam\u0131yorum. \u00c7\u00fcnk\u00fc deneyin amac\u0131 neydi ki?"\u201d<\/p>\n\n\n\n

Denemeler, \u00fcr\u00fcnlerin fayda g\u00f6sterme olas\u0131l\u0131\u011f\u0131n\u0131 en \u00fcst d\u00fczeye \u00e7\u0131karmak i\u00e7in tasarlanm\u0131\u015ft\u0131r.<\/p>\n\n\n\n

\"Ve e\u011fer bo\u015fsa, bo\u015ftur\" dedi.<\/strong><\/p>\n\n\n\n

H\u0131zland\u0131r\u0131lm\u0131\u015f Onay Yolu<\/h2>\n\n\n\n

FDA, son y\u0131llarda \u00e7ok say\u0131da vasat ilac\u0131n onay\u0131n\u0131 ald\u0131. Ross'un ortak yazarl\u0131\u011f\u0131n\u0131 yapt\u0131\u011f\u0131 bir ara\u015ft\u0131rmaya g\u00f6re, 2018 ile 2021 y\u0131llar\u0131 aras\u0131nda onaylanan ila\u00e7lar\u0131n 10%'den fazlas\u0131, \u00f6nemli bir denemede en az bir birincil sonlan\u0131m noktas\u0131n\u0131 kar\u015f\u0131layamayan bir ila\u00e7la ilgiliydi. >> Makaleyi Oku<\/a><\/p>\n\n\n\n

Bir ilac\u0131n faydas\u0131na dair net bir kan\u0131t olmadan onaylanmas\u0131, g\u00fcvenlik ve maliyet a\u00e7\u0131s\u0131ndan \u00e7e\u015fitli riskleri beraberinde getirir.<\/p>\n\n\n\n

\u201c"Bu \u00e7ok pahal\u0131 gen terapileri, biyolojik ila\u00e7lar ve di\u011fer \u00f6zel ila\u00e7lar\u0131n baz\u0131lar\u0131 aileleri iflasa s\u00fcr\u00fckleyebilir," dedi. Daha geni\u015f halk da, daha y\u00fcksek sigorta primleri ve di\u011fer maliyetlerle mali y\u00fck\u00fcn bir k\u0131sm\u0131n\u0131 \u00fcstleniyor.<\/p>\n\n\n\n

Hastalar\u0131 korumak i\u00e7in daha s\u0131k\u0131 FDA standartlar\u0131na ihtiya\u00e7 oldu\u011funu s\u00f6yledi.<\/p>\n\n\n\n

\u201cRoss, "Genel hissiyat\u0131m \u015fu: E\u011fer bir toplum olarak, \u00fcr\u00fcnlerin hastalara daha erken ula\u015fabilmesi i\u00e7in daha az kesin kan\u0131tla onaylanmas\u0131na izin vereceksek, \u015firketlerin onaylay\u0131c\u0131 denemeleri makul bir s\u00fcre i\u00e7inde piyasaya s\u00fcr\u00fcld\u00fckten sonra tamamlamalar\u0131n\u0131 sa\u011flamak i\u00e7in daha iyi bir i\u015f \u00e7\u0131karmal\u0131y\u0131z. B\u00f6ylece, \u00fcr\u00fcnlerin i\u015fe yarad\u0131\u011f\u0131n\u0131 ve hastalar\u0131n paralar\u0131n\u0131 bo\u015fa harcamad\u0131\u011f\u0131n\u0131 biliriz," dedi.<\/p>\n\n\n\n

Bu Sayfay\u0131 Takip Edin<\/strong> >>> Duchenne i\u00e7in T\u00fcm Klinik Ara\u015ft\u0131rmalar<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Do gene therapies, marketed for millions of dollars, and exon-skipping therapies administered monthly and sold for thousands of dollars, actually work well? Families campaigning under intense stress to access the therapies hope that DMD therapies will positively impact their children’s lives, but the data is less promising. It might have been the end of the […]<\/p>\n","protected":false},"author":1,"featured_media":5142,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[137,33,178,88,61,65],"class_list":["post-5141","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-clinical-trial","tag-elevidys","tag-exon-skipping-therapies","tag-fda","tag-gene-therapy","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5141","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=5141"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5141\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5142"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5141"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5141"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5141"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}