{"id":8153,"date":"2026-06-24T15:04:11","date_gmt":"2026-06-24T12:04:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8153"},"modified":"2026-06-24T15:51:29","modified_gmt":"2026-06-24T12:51:29","slug":"regenxbio-completes-rgx-202-confirmatory-study-for-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/regenxbio-completes-rgx-202-confirmatory-study-for-duchenne-muscular-dystrophy\/","title":{"rendered":"REGENXBIO, Duchenne Kas Distrofisi \u0130\u00e7in RGX-202 Do\u011frulama \u00c7al\u0131\u015fmas\u0131n\u0131 Tamamlad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">REGENXBIO, Duchenne Musk\u00fcler Distrofisi (DMD) i\u00e7in geli\u015ftirdi\u011fi deneysel gen terapisi RGX-202'nin do\u011frulama \u00e7al\u0131\u015fmas\u0131nda hasta dozlamas\u0131n\u0131n tamamland\u0131\u011f\u0131n\u0131 duyurdu. Bu \u00f6nemli kilometre ta\u015f\u0131, \u015firketin tescil geli\u015ftirme program\u0131n\u0131n tamamlanmas\u0131n\u0131 i\u015faret ediyor ve 2026 y\u0131l\u0131n\u0131n \u00fc\u00e7\u00fcnc\u00fc \u00e7eyre\u011finde ABD G\u0131da ve \u0130la\u00e7 \u0130daresi'ne (FDA) yap\u0131lmas\u0131 planlanan Biyolojik \u00dcr\u00fcn Lisans Ba\u015fvurusunu (BLA) destekliyor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, onaylanmas\u0131 halinde RGX-202&#039;nin Duchenne kas distrofisi olan ki\u015filer i\u00e7in kullan\u0131ma sunulacak yeni gen terapilerinden biri olabilece\u011fini \u00f6ng\u00f6r\u00fcyor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RGX-202 Geli\u015ftirme Program\u0131 Planlanandan \u00d6nce Tamamland\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">REGENXBIO&#039;nun a\u00e7\u0131klamas\u0131na g\u00f6re, hem hastalardan hem de klinik ara\u015ft\u0131rmac\u0131lardan gelen yo\u011fun ilgi nedeniyle do\u011frulama \u00e7al\u0131\u015fmas\u0131na kay\u0131t ve dozlama i\u015flemleri beklenenden daha erken tamamland\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00e7al\u0131\u015fman\u0131n tamamlanmas\u0131, \u015firketin FDA'n\u0131n h\u0131zland\u0131r\u0131lm\u0131\u015f onay yolu kapsam\u0131nda planlanan BLA ba\u015fvurusunu yapmas\u0131na olanak tan\u0131yor. Daha fazla bilgi i\u00e7in: <a href=\"https:\/\/dmdwarrior.com\/tr\/biologics-license-application-bla\/\" target=\"_blank\" data-type=\"post\" data-id=\"5027\" rel=\"noreferrer noopener\">FDA \u0130la\u00e7 Onaylar\u0131nda BLA Ne Anlama Geliyor?<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\">H\u0131zland\u0131r\u0131lm\u0131\u015f Onay S\u00fcreci Nedir?<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">H\u0131zland\u0131r\u0131lm\u0131\u015f onay s\u00fcreci, ciddi hastal\u0131klar i\u00e7in umut vadeden tedavilerin hastalara daha h\u0131zl\u0131 ula\u015fmas\u0131na yard\u0131mc\u0131 olmak \u00fczere tasarlanm\u0131\u015ft\u0131r. Bu s\u00fcre\u00e7te, tedaviler, klinik fayday\u0131 makul bir \u015fekilde \u00f6ng\u00f6rebilecek biyobelirte\u00e7ler gibi dolayl\u0131 g\u00f6stergelere dayanarak onay alabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">REGENXBIO, mikrodistrofin ekspresyon verileri, fonksiyonel sonu\u00e7lar ve g\u00fcvenlik profili temelinde RGX-202&#039;nin bu gereksinimleri kar\u015f\u0131lad\u0131\u011f\u0131na inanmaktad\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Planlanan FDA ba\u015fvurusu 2026 y\u0131l\u0131n\u0131n 3. \u00e7eyre\u011finde yap\u0131lacak<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">REGENXBIO, BLA ba\u015fvurusunu 2026 y\u0131l\u0131n\u0131n \u00fc\u00e7\u00fcnc\u00fc \u00e7eyre\u011finde ba\u015flatmay\u0131 planl\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ba\u015fvurunun a\u015fa\u011f\u0131daki unsurlar\u0131 i\u00e7ermesi beklenmektedir:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>AFFINITY DUCHENNE\u00ae temel ve do\u011frulama \u00e7al\u0131\u015fmalar\u0131na kat\u0131lan 63 kat\u0131l\u0131mc\u0131dan elde edilen g\u00fcvenlik verileri.<\/li>\n\n\n\n<li>Temel \u00e7al\u0131\u015fmaya kat\u0131lan 30 kat\u0131l\u0131mc\u0131dan elde edilen etkinlik verileri.<\/li>\n\n\n\n<li>\u00c7al\u0131\u015fman\u0131n kilit kat\u0131l\u0131mc\u0131lar\u0131n\u0131n en az yar\u0131s\u0131 i\u00e7in on iki ayl\u0131k fonksiyonel de\u011ferlendirme sonu\u00e7lar\u0131.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">FDA incelemesi beklendi\u011fi gibi ilerlerse, \u015firket RGX-202&#039;nin 2027&#039;nin ikinci yar\u0131s\u0131nda onay alabilece\u011fine inan\u0131yor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RGX-202 Temel \u00c7al\u0131\u015fmas\u0131n\u0131n Ba\u015fl\u0131ca Sonu\u00e7lar\u0131<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Yak\u0131n zamanda a\u00e7\u0131klanan \u00f6nemli \u00e7al\u0131\u015fma verileri, RGX-202 i\u00e7in umut verici sonu\u00e7lar g\u00f6sterdi.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Birincil Hedef Ba\u015far\u0131yla Elde Edildi<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">RGX-202, birincil hedefine ula\u015ft\u0131; hastalar\u0131n y\u00fczde 93'\u00fcnden fazlas\u0131, tedaviden 12 hafta sonra en az y\u00fczde 10 mikrodistrofin ekspresyonuna ula\u015ft\u0131. Daha Fazlas\u0131n\u0131 Okuyun: <a href=\"https:\/\/dmdwarrior.com\/tr\/rgx-202-phase-3-affinity-duchenne-results-released-by-regenxbio\/\" target=\"_blank\" rel=\"noreferrer noopener\">RGX-202, NSAA&#039;da art\u0131\u015f g\u00f6steriyor ancak \u00f6nemli biyobelirte\u00e7 verileri eksik.<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Mikrodistrofin, gen terapilerinin kas h\u00fccrelerinde \u00fcretmeyi hedefledi\u011fi distrofin proteininin k\u0131salt\u0131lm\u0131\u015f bir versiyonudur. Distrofin eksikli\u011fi, Duchenne Musk\u00fcler Distrofisinin temel nedenidir.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Fonksiyonel Sonu\u00e7larda \u0130yile\u015fmeler<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">12 ayl\u0131k takip de\u011ferlendirmelerini tamamlayan hastalar aras\u0131nda ara\u015ft\u0131rmac\u0131lar, \u00e7e\u015fitli fonksiyonel \u00f6l\u00e7\u00fcmlerde anlaml\u0131 iyile\u015fmeler g\u00f6zlemledi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bunlar aras\u0131nda \u015funlar yer al\u0131yordu:<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Zamanl\u0131 fonksiyon testleri<\/li>\n\n\n\n<li><a href=\"https:\/\/dmdwarrior.com\/tr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"4959\" rel=\"noreferrer noopener\">North Star Ambulatory Assessment (NSAA) puanlar\u0131<\/a><\/li>\n\n\n\n<li>Genel fiziksel performans g\u00f6stergeleri<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, ba\u015flang\u0131\u00e7 \u00f6l\u00e7\u00fcmlerine k\u0131yasla \u00f6nemli bir iyile\u015fme kaydetti\u011fini bildirdi.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">Mikrodistrofin ve Fonksiyon Aras\u0131nda G\u00fc\u00e7l\u00fc Korelasyon<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">REGENXBIO ayr\u0131ca, 12. haftadaki mikrodistrofin ekspresyonu ile tedaviden bir y\u0131l sonra g\u00f6zlemlenen fonksiyonel iyile\u015fmeler aras\u0131nda g\u00fc\u00e7l\u00fc bir ili\u015fki oldu\u011funu bildirdi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu bulgu, mikrodistrofinin Duchenne hastalar\u0131nda uzun vadeli klinik fayday\u0131 tahmin etmeye yard\u0131mc\u0131 olabilecek bir g\u00f6sterge olarak kullan\u0131m\u0131n\u0131 desteklemektedir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">RGX-202&#039;nin G\u00fcvenlik Profili<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, RGX-202&#039;nin \u00e7al\u0131\u015fma boyunca genel olarak iyi tolere edildi\u011fini belirtti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ara\u015ft\u0131rmac\u0131lar, REGENXBIO&#039;nun RGX-202&#039;yi halihaz\u0131rda geli\u015ftirilmekte olan di\u011fer potansiyel tedavi se\u00e7eneklerinden ay\u0131ran olumlu bir g\u00fcvenlik profili bildirdi\u011fini a\u00e7\u0131klad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Hastalar\u0131n takibi devam ettik\u00e7e, uzun vadeli g\u00fcvenlik konusunda ek bilgiler elde etmek i\u00e7in izlemeye devam edilecektir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">REGENXBIO Potansiyel Ticari Lansmana Haz\u0131rlan\u0131yor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Olas\u0131 bir FDA onay\u0131 beklentisiyle, REGENXBIO \u00fcretim ve ticari altyap\u0131s\u0131n\u0131 haz\u0131rlamaya \u015fimdiden ba\u015flad\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u015eirket, gelecekteki ticari tedarik i\u00e7in tasarlanan \u00fcretimin ge\u00e7en y\u0131l ba\u015flad\u0131\u011f\u0131 Maryland, Rockville&#039;deki \u00dcretim \u0130novasyon Merkezi&#039;ni kullan\u0131yor.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu \u00fcretim haz\u0131rl\u0131\u011f\u0131, RGX-202&#039;nin d\u00fczenleyici onay almas\u0131 durumunda daha geni\u015f hasta eri\u015fimini desteklemeye yard\u0131mc\u0131 olabilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Bu, Duchenne Hastal\u0131\u011f\u0131 Olan Aileler \u0130\u00e7in Ne Anlama Geliyor?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Do\u011frulay\u0131c\u0131 \u00e7al\u0131\u015fman\u0131n tamamlanmas\u0131, RGX-202 ve Duchenne toplulu\u011fu i\u00e7in \u00f6nemli bir ilerlemeyi temsil etmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">2026&#039;n\u0131n \u00fc\u00e7\u00fcnc\u00fc \u00e7eyre\u011finde yap\u0131lmas\u0131 planlanan FDA ba\u015fvurusu ve 2027&#039;nin ikinci yar\u0131s\u0131nda beklenen olas\u0131 onay karar\u0131yla birlikte, Duchenne kas distrofisi olan aileler yakla\u015fan d\u00fczenleyici geli\u015fmeleri yak\u0131ndan takip edecekler.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Onaylanmas\u0131 halinde, RGX-202, Duchenne Musk\u00fcler Distrofisi ile ya\u015fayan hastalar i\u00e7in ek bir gen terapisi se\u00e7ene\u011fi haline gelerek tedavi se\u00e7eneklerini geni\u015fletebilir ve potansiyel olarak uzun vadeli sonu\u00e7lar\u0131 iyile\u015ftirebilir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">S\u0131k\u00e7a Sorulan Sorular (SSS)<\/h2>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list\">\n<div id=\"faq-question-1782302007464\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">RGX-202 nedir?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>RGX-202, REGENXBIO taraf\u0131ndan Duchenne Musk\u00fcler Distrofisi (DMD) tedavisi i\u00e7in geli\u015ftirilen deneysel bir gen terapisidir.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1782302015108\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">REGENXBIO, RGX-202&#039;yi FDA&#039;ye ne zaman sunacak?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u015eirket, Biyolojik \u00dcr\u00fcn Lisans Ba\u015fvurusunu (BLA) 2026 y\u0131l\u0131n\u0131n \u00fc\u00e7\u00fcnc\u00fc \u00e7eyre\u011finde ba\u015flatmay\u0131 planl\u0131yor.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1782302022653\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u00c7al\u0131\u015fman\u0131n temel sonu\u00e7lar\u0131 nelerdi?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>Hastalar\u0131n y\u00fczde 93'\u00fcnden fazlas\u0131 12. haftada en az y\u00fczde 10 mikrodistrofin ekspresyonuna ula\u015ft\u0131 ve erken d\u00f6nem fonksiyonel veriler bir y\u0131l sonra anlaml\u0131 iyile\u015fmeler g\u00f6sterdi.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1782302027017\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">RGX-202 ne zaman onaylanabilir?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>REGENXBIO, FDA inceleme s\u00fcrecinin beklendi\u011fi gibi ilerlemesi durumunda RGX-202&#039;nin 2027 y\u0131l\u0131n\u0131n ikinci yar\u0131s\u0131nda onay alabilece\u011fine inan\u0131yor.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1782302033436\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">BLA paketine ka\u00e7 hasta dahil edilmi\u015ftir?<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>Planlanan ba\u015fvurunun, temel \u00e7al\u0131\u015fma kapsam\u0131ndaki 63 kat\u0131l\u0131mc\u0131dan elde edilen g\u00fcvenlik verilerini ve 30 kat\u0131l\u0131mc\u0131dan elde edilen etkinlik verilerini i\u00e7ermesi bekleniyor.<\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>REGENXBIO has announced the completion of patient dosing in the confirmatory study of RGX-202, its investigational gene therapy for Duchenne Muscular Dystrophy (DMD). This important milestone marks the completion of the company&#8217;s registrational development program and supports a planned Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) in the third [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8158,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[525,88,652,61,107,108],"class_list":["post-8153","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-affinity-duchenne","tag-fda","tag-fda-drug-approval","tag-gene-therapy","tag-regenxbio","tag-rgx-202"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8153","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=8153"}],"version-history":[{"count":3,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8153\/revisions"}],"predecessor-version":[{"id":8157,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/8153\/revisions\/8157"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/8158"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=8153"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=8153"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=8153"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}