{"id":4252,"date":"2025-07-29T11:18:46","date_gmt":"2025-07-29T08:18:46","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4252"},"modified":"2025-07-29T19:56:40","modified_gmt":"2025-07-29T16:56:40","slug":"metriopharm-mp1032-receives-ema-orphan-drug-designation-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/metriopharm-mp1032-receives-ema-orphan-drug-designation-in-duchenne-muscular-dystrophy\/","title":{"rendered":"MetrioPharm&#039;\u0131n MP1032&#039;si Duchenne Kas Distrofisi&#039;nde EMA Yetim \u0130la\u00e7 Tan\u0131m\u0131n\u0131 Ald\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\">MetrioPharm, bug\u00fcn Avrupa \u0130la\u00e7 Ajans\u0131&#039;n\u0131n, Duchenne kas distrofisi (DMD) hastal\u0131\u011f\u0131ndan etkilenen \u00e7ocuklar\u0131n tedavisi i\u00e7in \u00f6nc\u00fc bile\u015fi\u011fi MP1032&#039;ye Yetim \u0130la\u00e7 Tan\u0131m\u0131 (ODD) verdi\u011fini duyurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu kilometre ta\u015f\u0131, ABD&#039;deki FDA&#039;nin ODD&#039;sini takip ediyor ve MP1032&#039;nin, osteoporoz, b\u00fcy\u00fcme gerili\u011fi ve metabolik sorunlar gibi ciddi yan etkilere neden olan y\u00fcksek doz kortikosteroidlere olan ba\u011f\u0131ml\u0131l\u0131\u011f\u0131 en aza indirebilecek, s\u0131n\u0131f\u0131nda ilk tedavi olma potansiyelini vurguluyor.<\/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=\"#key-findings-of-metrio-pharm-mp-1032\">MetrioPharm MP1032&#039;nin Temel Bulgular\u0131<\/a><\/li><li><a href=\"#what-is-metrio-pharm-mp-1032\">MetrioPharm MP1032 nedir?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"key-findings-of-metrio-pharm-mp-1032\">MetrioPharm MP1032&#039;nin Temel Bulgular\u0131<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Avrupa \u0130la\u00e7 Ajans\u0131, Duchenne Musk\u00fcler Distrofi (DMD) hastal\u0131\u011f\u0131nda kar\u015f\u0131lanmam\u0131\u015f \u00f6nemli bir t\u0131bbi ihtiyac\u0131n giderilmesinde \u00f6nc\u00fc bile\u015fik MP1032&#039;nin potansiyelini kabul ediyor<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Bu tan\u0131mlama, daha g\u00fcvenli uzun vadeli tedavinin \u00f6n\u00fcn\u00fc a\u00e7\u0131yor<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>MP1032, normal h\u00fccresel s\u00fcre\u00e7leri bozmadan kas fonksiyonunu koruma ve iltihab\u0131 azaltma yetene\u011fini g\u00f6stermi\u015ftir<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">EMA&#039;nin ODD&#039;si, EMA&#039;den protokol deste\u011fi, \u00fccret muafiyetleri ve onay sonras\u0131nda AB&#039;de 12 y\u0131la kadar pazar m\u00fcnhas\u0131rl\u0131\u011f\u0131 (DMD&#039;nin pediatrik odakl\u0131 yakla\u015f\u0131m\u0131 nedeniyle 10 y\u0131ldan uzat\u0131lm\u0131\u015ft\u0131r) gibi \u00f6nemli te\u015fvikler sunmaktad\u0131r. Bu d\u00fczenleyici do\u011frulama, MP1032&#039;nin normal h\u00fccresel s\u00fcre\u00e7leri bozmadan kas fonksiyonunu koruma ve iltihab\u0131 azaltma yetene\u011fini g\u00f6steren ikna edici klinik \u00f6ncesi verilere dayanmaktad\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">CEO Thomas Christ\u00e9ly, &quot;FDA ve EMA&#039;den gelen bu ikili ODD ve FDA&#039;den al\u0131nan Nadir Pediatrik Hastal\u0131k Tan\u0131m\u0131, MetrioPharm ve DMD toplulu\u011fu i\u00e7in \u00e7\u0131\u011f\u0131r a\u00e7\u0131c\u0131 nitelikte,&quot; dedi. &quot;MP1032&#039;nin benzersiz mekanizmas\u0131 -a\u015f\u0131r\u0131 aktif ba\u011f\u0131\u015f\u0131kl\u0131k h\u00fccrelerinde mitokondriyal ROS&#039;u mod\u00fcle etmek- kortikosteroid dozlar\u0131n\u0131 ve yan etkilerini azaltarak uzun vadeli DMD y\u00f6netimini d\u00f6n\u00fc\u015ft\u00fcrebilir. MP1032&#039;yi Faz II denemelerine do\u011fru h\u0131zland\u0131r\u0131yor, hasta gruplar\u0131yla i\u015f birli\u011fi yap\u0131yor ve daha g\u00fcvenli se\u00e7eneklere ihtiya\u00e7 duyan \u00e7ocuklara sunmak i\u00e7in ortakl\u0131klar ara\u015ft\u0131r\u0131yoruz. DMD&#039;nin \u00f6tesinde, platformumuzun konak odakl\u0131 yakla\u015f\u0131m\u0131, HERA taraf\u0131ndan finanse edilen COVID-19 Faz IIa \u00e7al\u0131\u015fmam\u0131z\u0131n da kan\u0131tlad\u0131\u011f\u0131 gibi, otoimm\u00fcn hastal\u0131klarda ve hatta pandemilerde bile umut vadediyor.&quot;<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-metrio-pharm-mp-1032\">MetrioPharm MP1032 nedir?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>MetrioPharm&#039;\u0131n ba\u015fl\u0131ca aday\u0131 MP1032, aktif ba\u011f\u0131\u015f\u0131kl\u0131k h\u00fccrelerinde meydana gelen merkezi, do\u011fal metabolik s\u00fcreci ele alarak aktif ba\u011f\u0131\u015f\u0131kl\u0131k h\u00fccrelerinin a\u015f\u0131r\u0131 tepkimelerini hedefleyen benzersiz bir k\u00fc\u00e7\u00fck molek\u00fcld\u00fcr.<\/strong> Bu h\u00fccrelerin mitokondrileri, enerji metabolizmalar\u0131n\u0131 art\u0131rarak bu aktivasyona tepki verir ve bu da a\u015f\u0131r\u0131 reaktif oksijen t\u00fcrlerinin (ROS) olu\u015fumuna yol a\u00e7ar. MP1032, normal h\u00fccre fonksiyonu i\u00e7in gerekli olan di\u011fer h\u00fccrelerin fizyolojik ROS seviyelerini etkilemeden a\u015f\u0131r\u0131 ROS&#039;u azaltan, s\u0131n\u0131f\u0131n\u0131n ilk anti-inflamatuar ilac\u0131d\u0131r ve m\u00fckemmel bir g\u00fcvenlik profiline sahiptir. Monoterapi olarak MP1032, kortikosteroidlere (kortizon bazl\u0131 terap\u00f6tikler) benzer terap\u00f6tik etkiler \u00fcretir, ancak ciddi yan etkilere neden olmaz. Geleneksel anti-inflamatuarlar\u0131n aksine, monoterapi olarak kortikosteroid faydalar\u0131n\u0131 taklit eder, ancak DMD, multipl skleroz, artrit ve daha fazlas\u0131n\u0131n klinik \u00f6ncesi modellerinde g\u00f6sterildi\u011fi gibi toksisitelerinden ka\u00e7\u0131n\u0131r.<\/p>","protected":false},"excerpt":{"rendered":"<p>MetrioPharm announced today that the European Medicines Agency has granted Orphan Drug Designation (ODD) to its lead compound, MP1032 for the treatment of children affected by Duchenne muscular dystrophy (DMD). This milestone follows the U.S. FDA&#8217;s ODD, underscoring MP1032&#8217;s potential as a first-in-class therapy that could minimize reliance on high-dose corticosteroids, which cause severe side [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4253,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[91,213,398,399,195],"class_list":["post-4252","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ema","tag-european-medicines-agency","tag-metriopharm","tag-mp1032","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4252","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=4252"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4252\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4253"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4252"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4252"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4252"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}