{"id":2609,"date":"2025-08-03T09:40:50","date_gmt":"2025-08-03T09:40:50","guid":{"rendered":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/fda-genehmigt-neues-medikament-gegen-phenylketonurie-pku\/"},"modified":"2025-08-03T09:40:50","modified_gmt":"2025-08-03T09:40:50","slug":"fda-genehmigt-neues-medikament-gegen-phenylketonurie-pku","status":"publish","type":"post","link":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/fda-genehmigt-neues-medikament-gegen-phenylketonurie-pku\/","title":{"rendered":"FDA genehmigt neues Medikament gegen Phenylketonurie (PKU)"},"content":{"rendered":"<p>Die US-Beh\u00f6rde <a href=\"https:\/\/www.fda.gov\/\" data-internallinksmanager029f6b8e52c=\"8\" title=\"fda\">FDA<\/a> hat ein neues Medikament namens Sephience\u2122 (Sepiapterin) f\u00fcr die seltene Stoffwechselerkrankung Hyperphenylalanin\u00e4mie bei Patienten mit sepiapterin-ansprechender Phenylketonurie (PKU) zugelassen. Das Medikament ist f\u00fcr Erwachsene und Kinder ab einem Monat geeignet und wird zusammen mit einer phenylalanin- (Phe) reduzierten Di\u00e4t eingesetzt. Phenylketonurie f\u00fchrt zu einer sch\u00e4dlichen Ansammlung von Phe im K\u00f6rper, was durch einen Gendefekt verursacht wird, der die Produktion eines Enzyms zur Abbau des Aminos\u00e4ure Phenylalanin beeintr\u00e4chtigt. Sephience kann laut Hersteller PTC Therapeutics die Phenylalanin-Werte im Blut senken. Die Zulassung basiert auf den positiven Ergebnissen der Phase-III-Studie APHENITY sowie einer Langzeitstudie, die die Wirksamkeit und Sicherheit des Medikaments best\u00e4tigten. Die Europ\u00e4ische Kommission hatte Sepiapterin bereits im vergangenen Monat f\u00fcr alle Altersgruppen und Schweregrade der PKU zugelassen. Weitere Zulassungsverfahren laufen in L\u00e4ndern wie Japan und Brasilien. Dr. Matthew Klein, CEO von PTC Therapeutics, betont: \u201eWir sind begeistert, diesen wichtigen Meilenstein f\u00fcr PKU-Patienten erreicht zu haben. Die Daten und unsere Erfahrung in der Behandlung seltener Erkrankungen positionieren Sephience als zuk\u00fcnftigen Standard in der PKU-Therapie.\u201c Die Krankheit betrifft weltweit etwa 58.000 Menschen und kann zu bleibenden geistigen Behinderungen, Krampfanf\u00e4llen, Entwicklungsverz\u00f6gerungen und Ged\u00e4chtnisverlust f\u00fchren.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Die US-Beh\u00f6rde FDA hat ein neues Medikament namens Sephience\u2122 (Sepiapterin) f\u00fcr die seltene Stoffwechselerkrankung Hyperphenylalanin\u00e4mie bei Patienten mit sepiapterin-ansprechender Phenylketonurie (PKU) zugelassen. Das Medikament ist f\u00fcr Erwachsene und Kinder ab einem Monat geeignet und wird zusammen mit einer phenylalanin- (Phe) reduzierten Di\u00e4t eingesetzt. Phenylketonurie f\u00fchrt zu einer sch\u00e4dlichen Ansammlung von Phe im K\u00f6rper, was durch [&hellip;]<\/p>\n","protected":false},"author":0,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"footnotes":""},"categories":[489],"tags":[],"class_list":["post-2609","post","type-post","status-publish","format-standard","hentry","category-pharmazeutische-nachrichten"],"_links":{"self":[{"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/posts\/2609","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/types\/post"}],"replies":[{"embeddable":true,"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/comments?post=2609"}],"version-history":[{"count":0,"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/posts\/2609\/revisions"}],"wp:attachment":[{"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/media?parent=2609"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/categories?post=2609"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/schloss-apotheke-kuemmersbruck.de\/produkte\/wp-json\/wp\/v2\/tags?post=2609"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}