{"id":3929,"date":"2025-06-17T16:48:33","date_gmt":"2025-06-17T13:48:33","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3929"},"modified":"2025-06-17T16:48:34","modified_gmt":"2025-06-17T13:48:34","slug":"capricor-therapeutics-announces-bmd-orphan-drug-designation-and-dmd-program-regulatory-development-for-deramiocel-cap-1002","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/capricor-therapeutics-announces-bmd-orphan-drug-designation-and-dmd-program-regulatory-development-for-deramiocel-cap-1002\/","title":{"rendered":"Capricor Therapeutics, Deramiocel (CAP-1002) i\u00e7in BMD Yetim \u0130la\u00e7 Tan\u0131m\u0131n\u0131 ve DMD Program\u0131 D\u00fczenleyici Geli\u015fimini Duyurdu"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Nadir hastal\u0131klar\u0131n tedavisi i\u00e7in d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc h\u00fccre ve ekzosom tabanl\u0131 tedaviler geli\u015ftiren bir biyoteknoloji \u015firketi olan Capricor Therapeutics, bug\u00fcn ABD G\u0131da ve \u0130la\u00e7 Dairesi&#039;nin (FDA) Becker Musk\u00fcler Distrofisi&#039;nin (BMD) potansiyel tedavisi i\u00e7in \u015firketin \u00f6nc\u00fc h\u00fccre tedavisi aday\u0131 olan Deramiocel&#039;ye (CAP-1002) Yetim \u0130la\u00e7 Tan\u0131m\u0131 verdi\u011fini duyurdu. Bu tan\u0131mlama, Capricor&#039;nin kas distrofisinin kardiyak ve iskelet komplikasyonlar\u0131n\u0131 hedefleyen tam entegre bir platformu ilerletmesi ve \u00f6nc\u00fc varl\u0131\u011f\u0131n\u0131n ticari potansiyelini geni\u015fletmesi nedeniyle stratejik konumunu g\u00fc\u00e7lendiriyor. \u2013 <a href=\"https:\/\/dmdwarrior.com\/tr\/what-is-deramiocel\/\">Deramiocel nedir? CAP-1002 ne i\u015fe yarar?<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Capricor Therapeutics Emin Ad\u0131mlarla \u0130lerliyor<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>ABD FDA, Deramiocel&#039;ye Becker Musk\u00fcler Distrofisi tedavisi i\u00e7in Yetim \u0130la\u00e7 Tan\u0131m\u0131 vererek, Capricor&#039;nin n\u00f6rom\u00fcsk\u00fcler hastal\u0131klardaki oda\u011f\u0131n\u0131 geni\u015fletiyor<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Capricor, ba\u015far\u0131l\u0131 FDA Lisans \u00d6ncesi denetimleri ard\u0131ndan Duchenne Musk\u00fcler Distrofisi'nde Deramiocel i\u00e7in Re\u00e7eteli \u0130la\u00e7 Kullan\u0131m \u00dccreti Yasas\u0131 (PDUFA) tarihi olan 31 A\u011fustos 2025'e do\u011fru yolda emin ad\u0131mlar \u00e7al\u0131\u015fmalar\u0131na devam ediyor.<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Becker Musk\u00fcler Distrofisi, Duchenne Musk\u00fcler Distrofisi (DMD) gibi, zamanla iskelet ve kalp kaslar\u0131nda \u00f6nemli bozulmaya yol a\u00e7an ilerleyici bir X&#039;e ba\u011fl\u0131 n\u00f6rom\u00fcsk\u00fcler hastal\u0131kt\u0131r. Deramiocel, hem BMD hem de DMD&#039;li hastalarda morbidite ve mortaliteye birincil katk\u0131da bulunan kardiyomiyopati de dahil olmak \u00fczere hastal\u0131k patolojisinin her iki y\u00f6n\u00fcn\u00fc ele almak i\u00e7in geli\u015ftirilmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Capricor&#039;nin DMD&#039;de Deramiocel i\u00e7in Biyolojik Lisans Ba\u015fvurusu (BLA) \u00f6ncelikli inceleme alt\u0131nda olup, PDUFA hedef eylem tarihi 31 A\u011fustos 2025&#039;tir.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/capricor-therapeutics-deramiocel-mid-cycle-review-meeting-with-fda\/\">Capricor Therapeutics \u0130nceleme Toplant\u0131s\u0131n\u0131n Tamamland\u0131\u011f\u0131n\u0131 Duyurdu<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel (CAP-1002), the company\u2019s lead cell therapy candidate, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation strengthens Capricor\u2019s strategic [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3931,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[361,90,86,87,89,85,117,362,88],"class_list":["post-3929","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-bmd","tag-cap-1002","tag-capricor","tag-capricor-therapeutics","tag-cardiomyopathy","tag-deramiocel","tag-dmd","tag-dmd-cardiomyopathy","tag-fda"],"subtitle":"Becker kas distrofisi i\u00e7in yetim ila\u00e7 stat\u00fcs\u00fc Capricor Therapeutics taraf\u0131ndan duyuruldu ve Duchenne kas distrofisi program\u0131 i\u00e7in d\u00fczenleyici ilerleme kaydedildi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3929","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=3929"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3929\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3931"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3929"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3929"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3929"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}