{"id":4182,"date":"2025-07-23T18:14:43","date_gmt":"2025-07-23T15:14:43","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4182"},"modified":"2025-07-24T15:39:40","modified_gmt":"2025-07-24T12:39:40","slug":"fda-designates-avidity-biosciences-delpacibart-zotadirsen-del-zota-as-a-breakthrough-therapy-for-the-treatment-of-dmd-in-individuals-with-mutations-that-allow-for-exon-44-skipping","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/fda-designates-avidity-biosciences-delpacibart-zotadirsen-del-zota-as-a-breakthrough-therapy-for-the-treatment-of-dmd-in-individuals-with-mutations-that-allow-for-exon-44-skipping\/","title":{"rendered":"FDA, Avidity Biosciences'\u0131n Delpacibart Zotadirsen'ini (del-zota), Ekzon 44 Atlamaya \u0130zin Veren Mutasyonlara Sahip Bireylerde DMD Tedavisi \u0130\u00e7in \u00c7\u0131\u011f\u0131r A\u00e7an Bir Terapi Olarak Belirledi"},"content":{"rendered":"

Avidity Biosciences bug\u00fcn ABD G\u0131da ve \u0130la\u00e7 Dairesi'nin (FDA) delpacibart zotadirsen'e (del-zota) Duchenne kas distrofisi (DMD) ve ekzon 44 atlamaya yatk\u0131n mutasyonlara sahip ki\u015filerde (DMD44) tedavi amac\u0131yla \u00c7\u0131\u011f\u0131r A\u00e7an Terapi unvan\u0131 verdi\u011fini duyurdu.<\/p>\n\n\n\n

Del-zota \u015fu anda DMD44 ile ya\u015fayan ki\u015filer i\u00e7in Faz 2 EXPLORE44 A\u00e7\u0131k Etiketli Uzatma (EXPLORE44-OLE\u2122) denemesinde de\u011ferlendiriliyor ve \u015firketin DMD i\u00e7in geli\u015ftirdi\u011fi \u00e7ok say\u0131da AOC'nin ilki.<\/p>\n\n\n\n

DMD, genellikle \u00e7ok gen\u00e7 ya\u015flarda ba\u015flayan distrofin proteininin kayb\u0131na ba\u011fl\u0131 ilerleyici kas hasar\u0131 ve g\u00fc\u00e7s\u00fczl\u00fc\u011f\u00fc ile karakterize nadir bir genetik rahats\u0131zl\u0131kt\u0131r. Del-zota, distrofin geninin 44. ekzonunu \u00f6zel olarak atlamak ve neredeyse tam uzunlukta distrofin \u00fcretimini sa\u011flamak i\u00e7in iskelet kas\u0131 ve kalp dokusuna fosforodiamidat morfolino oligomerleri (PMO'lar) iletmek \u00fczere tasarlanm\u0131\u015ft\u0131r.<\/strong> - Devam\u0131n\u0131 oku: Avidity Biosciences, Distrofin'de Tutarl\u0131, \u0130statistiksel Olarak Anlaml\u0131 \u0130yile\u015fmeler G\u00f6steren Pozitif Topline Del-zota Verilerini Duyurdu<\/a> \u2013<\/p>\n\n\n\n

Avidity Ba\u015f T\u0131p Sorumlusu Dr. Steve Hughes, "\u00c7\u0131\u011f\u0131r A\u00e7an Terapi unvan\u0131, FDA'nin del-zota'nin DMD44'\u00fcn alt\u0131nda yatan nedeni ele alma konusundaki \u00f6nemli potansiyeline ve DMD toplulu\u011funa yenilik\u00e7i tedavi se\u00e7enekleri sunma konusundaki acil ihtiyac\u0131na olan takdirini daha da vurguluyor," dedi. "Faz 1\/2 EXPLORE44 denemesinde distrofin de dahil olmak \u00fczere bir\u00e7ok biyobelirte\u00e7te g\u00f6rd\u00fc\u011f\u00fcm\u00fcz dikkat \u00e7ekici ve tutarl\u0131 iyile\u015fmelerle, del-zota'yi DMD44 ile ya\u015fayan insanlara m\u00fcmk\u00fcn olan en k\u0131sa s\u00fcrede ula\u015ft\u0131rmaya odaklan\u0131yoruz ve 2025 y\u0131l\u0131 sonunda planlanan BLA ba\u015fvurumuz i\u00e7in do\u011fru yolday\u0131z."<\/p>\n\n\n\n

DMD44 ile ya\u015fayan bireylerde tamamlanan Faz 1\/2 EXPLORE44\u00ae \u00e7al\u0131\u015fmas\u0131nda, del-zota, ekzon atlamada istatistiksel olarak anlaml\u0131 art\u0131\u015flar, distrofin \u00fcretiminde \u00f6nemli bir art\u0131\u015f, kreatin kinaz seviyelerinde normale yak\u0131n anlaml\u0131 ve s\u00fcrekli bir d\u00fc\u015f\u00fc\u015f ve tutarl\u0131 olumlu g\u00fcvenlik ve tolere edilebilirlik g\u00f6stermi\u015ftir. Avidity, devam eden ve tam kat\u0131l\u0131mc\u0131l\u0131 Faz 2 EXPLORE44-OLE \u00e7al\u0131\u015fmas\u0131ndan elde edilen temel ve i\u015flevsel verileri 2025'in d\u00f6rd\u00fcnc\u00fc \u00e7eyre\u011finde sunmay\u0131 planlamaktad\u0131r.<\/p>\n\n\n\n

Daha fazla bilgi edin: Duchenne Hastal\u0131\u011f\u0131nda Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n

\u015eirket, 2025 y\u0131l sonu i\u00e7in planlanan BLA ba\u015fvurusunu ger\u00e7ekle\u015ftirme yolunda ilerlemektedir. Avidity'nin, FDA onay\u0131n\u0131n ard\u0131ndan DMD44'te del-zota'nin olas\u0131 ABD lansman\u0131 i\u00e7in ticari haz\u0131rl\u0131klar\u0131 devam etmektedir. Del-zota'n\u0131n beklenen lansman\u0131, Avidity'nin miyotonik distrofi tip 1'de (DM1) del-desiran ve fasiyoskapulohumeral kas distrofisinde (FSHD) del-brax i\u00e7in ek n\u00f6rom\u00fcsk\u00fcler programlar\u0131n\u0131n potansiyel ard\u0131\u015f\u0131k lansmanlar\u0131n\u0131n temelini olu\u015fturmaktad\u0131r.<\/p>\n\n\n\n

\u00c7\u0131\u011f\u0131r A\u00e7an Tedavi tan\u0131m\u0131, ciddi bir rahats\u0131zl\u0131\u011f\u0131 tedavi etmeyi ama\u00e7layan ila\u00e7lar\u0131n geli\u015ftirilmesini ve g\u00f6zden ge\u00e7irilmesini h\u0131zland\u0131rmak i\u00e7in tasarlanm\u0131\u015f bir s\u00fcre\u00e7tir ve \u00f6n klinik kan\u0131tlar, ilac\u0131n klinik olarak anlaml\u0131 bir veya birden fazla son noktada mevcut tedaviye k\u0131yasla \u00f6nemli bir geli\u015fme g\u00f6sterebilece\u011fini g\u00f6stermektedir.<\/p>\n\n\n\n

del-zota, \u00c7\u0131\u011f\u0131r A\u00e7an Tedavi unvan\u0131n\u0131 almas\u0131n\u0131n yan\u0131 s\u0131ra, daha \u00f6nce FDA ve Avrupa \u0130la\u00e7 Ajans\u0131 (EMA) taraf\u0131ndan Yetim unvan\u0131 ve FDA taraf\u0131ndan DMD44 tedavisi i\u00e7in Nadir Pediatrik Hastal\u0131k ve H\u0131zl\u0131 Tedavi unvanlar\u0131n\u0131 alm\u0131\u015ft\u0131.<\/p>\n\n\n\n

Daha fazla bilgi edin: Duchenne Musk\u00fcler Distrofi Tedavisinde Yakla\u015fan Ekzon 44 Atlama Terapileri<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Avidity Biosciences today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for the treatment of Duchenne muscular dystrophy (DMD) in people living with mutations amenable to exon 44 skipping (DMD44). Del-zota is currently being assessed in the Phase 2 EXPLORE44 Open-Label Extension (EXPLORE44-OLE\u2122) trial for […]<\/p>\n","protected":false},"author":1,"featured_media":4185,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,397,141,127,246,299,359],"class_list":["post-4182","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44","tag-full-length-dystrophin"],"subtitle":"Avidity Biosciences, FDA'n\u0131n, ekzon 44 atlamaya uygun olan hastalarda Duchenne kas distrofisi (DMD) i\u00e7in \u00e7\u0131\u011f\u0131r a\u00e7an bir tedavi olarak delpacibart zotadirsen'i (del-zota) belirledi\u011fini bildirdi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4182","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=4182"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4182\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4185"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4182"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4182"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4182"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}