{"id":2923,"date":"2025-03-29T00:23:22","date_gmt":"2025-03-28T23:23:22","guid":{"rendered":"https:\/\/www.cisztasfibrozis.hu\/?p=2923"},"modified":"2025-03-29T00:25:08","modified_gmt":"2025-03-28T23:25:08","slug":"az-europai-gyogyszerugynokseg-javasolja-a-cisztas-fibrozis-gyogyszerek-kombinaciojanak-terapias-indikaciojanak-kiterjeszteset-ritka-mutacioval-rendelkezo-betegek-kezelesere","status":"publish","type":"post","link":"https:\/\/www.cisztasfibrozis.hu\/index.php\/2025\/03\/29\/az-europai-gyogyszerugynokseg-javasolja-a-cisztas-fibrozis-gyogyszerek-kombinaciojanak-terapias-indikaciojanak-kiterjeszteset-ritka-mutacioval-rendelkezo-betegek-kezelesere\/","title":{"rendered":"Az Eur\u00f3pai Gy\u00f3gyszer\u00fcgyn\u00f6ks\u00e9g javasolja a ciszt\u00e1s fibr\u00f3zis gy\u00f3gyszerek kombin\u00e1ci\u00f3j\u00e1nak ter\u00e1pi\u00e1s indik\u00e1ci\u00f3j\u00e1nak kiterjeszt\u00e9s\u00e9t ritka mut\u00e1ci\u00f3val rendelkez\u0151 betegek kezel\u00e9s\u00e9re"},"content":{"rendered":"\n<p>Az <a href=\"https:\/\/www.ema.europa.eu\/en\/homepage\" target=\"_blank\" rel=\"noopener\">Eur\u00f3pai Gy\u00f3gyszer\u00fcgyn\u00f6ks\u00e9g (EMA)<\/a> hum\u00e1n gy\u00f3gyszerekkel foglalkoz\u00f3 bizotts\u00e1ga <a href=\"https:\/\/www.ema.europa.eu\/en\/committees\/committee-medicinal-products-human-use-chmp\" target=\"_blank\" rel=\"noopener\">(CHMP)<\/a> pozit\u00edv aj\u00e1nl\u00e1st tett a <a href=\"https:\/\/ec.europa.eu\/health\/documents\/community-register\/2023\/20230717159650\/anx_159650_hu.pdf\" target=\"_blank\" rel=\"noopener\">Kaftrio (ivakaftor\/tezakaftor\/elexakaftor)<\/a> \u00e9s a Kalydeco (ivakaftor) kombin\u00e1ci\u00f3j\u00e1nak ter\u00e1pi\u00e1s indik\u00e1ci\u00f3j\u00e1nak kiterjeszt\u00e9s\u00e9re. Ez a v\u00e1ltoztat\u00e1s lehet\u0151v\u00e9 tenn\u00e9 a 12 \u00e9vesn\u00e9l id\u0151sebb, ritka mut\u00e1ci\u00f3val rendelkez\u0151 ciszt\u00e1s fibr\u00f3zisos (CF) betegek sz\u00e1m\u00e1ra a kezel\u00e9st, akikn\u00e9l a CFTR g\u00e9nben legal\u00e1bb egy olyan mut\u00e1ci\u00f3 tal\u00e1lhat\u00f3, amelyre a kombin\u00e1ci\u00f3 hat\u00e1sos.<\/p>\n\n\n\n<p>A ciszt\u00e1s fibr\u00f3zis egy s\u00falyos, \u00f6r\u00f6kletes betegs\u00e9g, amely f\u0151leg a t\u00fcd\u0151t \u00e9s a em\u00e9szt\u0151rendszert \u00e9rinti. A betegs\u00e9g m\u00f6g\u00f6tt a CFTR feh\u00e9rje m\u0171k\u00f6d\u00e9szavar \u00e1ll, amely a test t\u00f6bb szerv\u00e9ben is probl\u00e9m\u00e1kat okoz. A Kaftrio \u00e9s Kalydeco kombin\u00e1ci\u00f3ja a hib\u00e1s CFTR feh\u00e9rje m\u0171k\u00f6d\u00e9s\u00e9t jav\u00edtja, ezzel cs\u00f6kkentve a t\u00fcneteket \u00e9s jav\u00edtva a betegek \u00e9letmin\u0151s\u00e9g\u00e9t.<\/p>\n\n\n\n<p>A CHMP pozit\u00edv v\u00e9lem\u00e9nye alapj\u00e1n a kombin\u00e1ci\u00f3 most m\u00e1r olyan betegek sz\u00e1m\u00e1ra is el\u00e9rhet\u0151 lehet, akikn\u00e9l kor\u00e1bban nem volt kezel\u00e9si lehet\u0151s\u00e9g a ritka mut\u00e1ci\u00f3k miatt. Ez a d\u00f6nt\u00e9s jelent\u0151s l\u00e9p\u00e9st jelent a CF-ben szenved\u0151 betegek sz\u00e1m\u00e1ra, k\u00fcl\u00f6n\u00f6sen azoknak, akiknek mut\u00e1ci\u00f3i eddig kev\u00e9sb\u00e9 voltak vizsg\u00e1ltak vagy kezelhet\u0151k.<\/p>\n\n\n\n<p>Az aj\u00e1nl\u00e1st most az Eur\u00f3pai Bizotts\u00e1g ellen\u0151rzi, amely d\u00f6nt a v\u00e9gleges enged\u00e9lyez\u00e9sr\u0151l az Eur\u00f3pai Uni\u00f3 tag\u00e1llamaiban. Ha j\u00f3v\u00e1hagyj\u00e1k, a kombin\u00e1ci\u00f3 haszn\u00e1lata b\u0151v\u00fclhet, \u00e9s t\u00f6bb beteg sz\u00e1m\u00e1ra ny\u00edlik meg a kezel\u00e9s lehet\u0151s\u00e9ge.<\/p>\n\n\n\n<p>A <a href=\"https:\/\/www.cf-europe.eu\" target=\"_blank\" rel=\"noopener\">CF Europe<\/a>, a ciszt\u00e1s fibr\u00f3zisban szenved\u0151 betegek eur\u00f3pai szervezete \u00fcdv\u00f6z\u00f6lte a CHMP d\u00f6nt\u00e9s\u00e9t, kiemelve, hogy ez \u00faj rem\u00e9nyt adhat azoknak a betegeknek, akik kor\u00e1bban korl\u00e1tozott ter\u00e1pi\u00e1s lehet\u0151s\u00e9gekkel rendelkeztek.<\/p>\n\n\n\n<figure class=\"wp-block-embed is-type-video is-provider-youtube wp-block-embed-youtube wp-embed-aspect-16-9 wp-has-aspect-ratio\"><div class=\"wp-block-embed__wrapper\">\n<iframe loading=\"lazy\" title=\"Audrey Chansard explains EMA&#039;s new CF treatment decision and next steps\" width=\"500\" height=\"281\" src=\"https:\/\/www.youtube.com\/embed\/dniYog9Am3o?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share\" referrerpolicy=\"strict-origin-when-cross-origin\" allowfullscreen><\/iframe>\n<\/div><\/figure>\n","protected":false},"excerpt":{"rendered":"<p>Az Eur\u00f3pai Gy\u00f3gyszer\u00fcgyn\u00f6ks\u00e9g (EMA) hum\u00e1n gy\u00f3gyszerekkel foglalkoz\u00f3 bizotts\u00e1ga (CHMP) pozit\u00edv aj\u00e1nl\u00e1st tett a Kaftrio (ivakaftor\/tezakaftor\/elexakaftor) \u00e9s a Kalydeco (ivakaftor) kombin\u00e1ci\u00f3j\u00e1nak ter\u00e1pi\u00e1s indik\u00e1ci\u00f3j\u00e1nak kiterjeszt\u00e9s\u00e9re. Ez a&#8230;<\/p>\n","protected":false},"author":1,"featured_media":2912,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[2,1],"tags":[],"class_list":["post-2923","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news","category-uncategorized","entry","double"],"_links":{"self":[{"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/posts\/2923","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/comments?post=2923"}],"version-history":[{"count":1,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/posts\/2923\/revisions"}],"predecessor-version":[{"id":2926,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/posts\/2923\/revisions\/2926"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/media\/2912"}],"wp:attachment":[{"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/media?parent=2923"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/categories?post=2923"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.cisztasfibrozis.hu\/index.php\/wp-json\/wp\/v2\/tags?post=2923"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}