{"id":1710,"date":"2014-06-30T20:12:28","date_gmt":"2014-06-30T19:12:28","guid":{"rendered":"http:\/\/slanedeti.sk\/cms\/?p=1710"},"modified":"2014-07-01T06:42:33","modified_gmt":"2014-07-01T05:42:33","slug":"prelom-v-liecbe-f508del","status":"publish","type":"post","link":"https:\/\/slanedeti.sk\/cms\/prelom-v-liecbe-f508del\/1710","title":{"rendered":"Prelom v lie\u010dbe f508del"},"content":{"rendered":"<p>Spolo\u010dnos\u0165 Vertex Pharmaceuticals zverejnila 24.j\u00fana<a href=\"http:\/\/investors.vrtx.com\/releasedetail.cfm?ReleaseID=856185\"> v\u00fdsledky tretej f\u00e1zy<\/a> klinick\u00fdch \u0161t\u00fadii kombinovanej terapie ivacaftoru (Kalydeco) a lumacaftoru (VX-809) u pacientov s dvomi k\u00f3piami naj\u010dastej\u0161ej CF mut\u00e1cie &#8211; F508del. V\u00fdsledky preuk\u00e1zali \u0161tatisticky v\u00fdznamn\u00e9 zlep\u0161enia p\u013e\u00facnych funkci\u00ed a \u010fal\u0161\u00edch sledovan\u00fdch parametrov. Priemern\u00e9 relat\u00edvne zlep\u0161enie p\u013e\u00facnych funkci\u00ed bolo o 4,3 a\u017e 6,7 %. Po\u010det exacerb\u00e1cii sa zn\u00ed\u017eil o 30 a\u017e 39 % a lie\u010dba tie\u017e viedla k miernemu zv\u00fd\u0161eniu body mass indexu. Lie\u010dba bola vo v\u0161eobecnosti dobre tolerovan\u00e1 a zo \u0161t\u00fadie kv\u00f4li ne\u017eiad\u00facim \u00fa\u010dinkom odst\u00fapilo iba 4,2 % pacientov. \u0160t\u00fadia (rozdelen\u00e1 na \u0161t\u00fadie TRAFFIC a TRANSPORT) prebiehala na pribli\u017ene 200 klinick\u00fdch pracovisk\u00e1ch v Severnej Amerike, Eur\u00f3pe a Austr\u00e1lii, pri\u010dom bolo do nej zapojen\u00fdch viac ako 1100 pacientov, vo veku nad 12 rokov, s dvomi F508del mut\u00e1ciami. Na z\u00e1klade t\u00fdchto v\u00fdsledkov pl\u00e1nuje Vertex predlo\u017ei\u0165 Americk\u00e9mu \u00faradu pre potraviny a lieky (FDA) \u017eiados\u0165 o uvedenie nov\u00e9ho lieku na trh, pri\u010dom schv\u00e1lenie je mo\u017en\u00e9 o\u010dak\u00e1va\u0165 v roku 2015.<\/p>\n<p>&nbsp;<\/p>\n<p><img loading=\"lazy\" decoding=\"async\" class=\"aligncenter size-full wp-image-1711\" src=\"https:\/\/slanedeti.sk\/cms\/wp-content\/uploads\/2014\/06\/kalydeco_vx809_tab.png\" alt=\"tabulka\" width=\"626\" height=\"213\" srcset=\"https:\/\/slanedeti.sk\/cms\/wp-content\/uploads\/2014\/06\/kalydeco_vx809_tab.png 626w, https:\/\/slanedeti.sk\/cms\/wp-content\/uploads\/2014\/06\/kalydeco_vx809_tab-300x102.png 300w\" sizes=\"auto, (max-width: 626px) 100vw, 626px\" \/><\/p>\n<p style=\"text-align: center;\"><em>Zhrnutie v\u00fdsledkov \u0161t\u00fadie pri prim\u00e1rnom vyhodnocovanom ukazovateli \u2013 FEV1 <a href=\"http:\/\/www.vrtx.com\/\">(www.vrtx.com)<\/a><\/em><\/p>\n<p>Ivacaftor a lumacaftor s\u00fa lie\u010div\u00e1 vyvinut\u00e9 spolo\u010dnos\u0165ou Vertex Pharmaceuticals, s finan\u010dnou podporou americkej Cystic Fibrosis Foundation a National Institutes of Health. Ivacaftor bol schv\u00e1len\u00fd (pod obchodn\u00fdm n\u00e1zvom Kalydeco) v roku 2012, ako prv\u00fd liek s kauz\u00e1lnym \u00fa\u010dinkom (ovplyv\u0148uj\u00facim z\u00e1kladn\u00fd defekt ochorenia) pri cystickej fibr\u00f3ze. P\u00f4vodne schv\u00e1lenou indik\u00e1ciou bola mut\u00e1cia G551D, vo febru\u00e1ri 2014 bolo pou\u017eitie<a href=\"http:\/\/investors.vrtx.com\/releasedetail.cfm?ReleaseID=827435\"> roz\u0161\u00edren\u00e9 na \u010fal\u0161\u00edch 8 mut\u00e1cii.<\/a> V\u0161etky mut\u00e1cie pre ktor\u00e9 bola doteraz schv\u00e1len\u00e1 indik\u00e1cia nie s\u00fa pr\u00edli\u0161 roz\u0161\u00edren\u00e9. Oproti tomu mut\u00e1cia F508del je v CF popul\u00e1ci\u00e1ch dominantnou a jej dve k\u00f3pie maj\u00fa desa\u0165tis\u00edce pacientov. Na z\u00e1klade v\u00fdsledkov klinickej \u0161t\u00fadie je re\u00e1lne o\u010dak\u00e1va\u0165, \u017ee na trhu bude v bl\u00edzkej dobe pre ve\u013ek\u00fa \u010das\u0165 CF pacientov doteraz naj\u00fa\u010dinnej\u0161\u00ed liek. Je to mo\u017en\u00e9 pova\u017eova\u0165 za jeden z historick\u00fdch prielomov v lie\u010dbe CF.<br \/>\nAkcie spolo\u010dnosti Vertex po zverejnen\u00ed ozn\u00e1menia st\u00fapli o 42%. Predpoklad\u00e1 sa, \u017ee celosvetov\u00fd predaj nov\u00e9ho lieku by mohol dosiahnu\u0165 3 miliardy dol\u00e1rov ro\u010dne. Ot\u00e1zna ost\u00e1va bud\u00faca cenov\u00e1 politika Vertexu, ke\u010f\u017ee doteraz boli n\u00e1klady na ro\u010dn\u00fa lie\u010dbu ivacaftorom okolo $ 300 000. Tak stanoven\u00e1 cena bola ter\u010dom v\u00fdraznej kritiky a ani v mnoh\u00fdch ekonomicky vyspel\u00fdch krajin\u00e1ch by pri podobne stanovenej cene financovanie lie\u010dby zo zdrojov zdravotn\u00e9ho poistenia nebolo mo\u017en\u00e9.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Spolo&#269;nos&#357; Vertex Pharmaceuticals zverejnila 24.j&uacute;na v&yacute;sledky tretej f&aacute;zy klinick&yacute;ch &scaron;t&uacute;dii kombinovanej terapie ivacaftoru (Kalydeco) a lumacaftoru (VX-809) u pacientov s dvomi k&oacute;piami naj&#269;astej&scaron;ej CF mut&aacute;cie &ndash; F508del. V&yacute;sledky preuk&aacute;zali &scaron;tatisticky v&yacute;znamn&eacute; zlep&scaron;enia p&#318;&uacute;cnych funkci&iacute; a &#271;al&scaron;&iacute;ch sledovan&yacute;ch parametrov. Priemern&eacute; relat&iacute;vne &hellip; <a href=\"https:\/\/slanedeti.sk\/cms\/prelom-v-liecbe-f508del\/1710\">Continued<\/a><\/p>\n","protected":false},"author":7,"featured_media":1714,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"kt_blocks_editor_width":"","footnotes":""},"categories":[3],"tags":[],"class_list":["post-1710","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-lieky-a-liecba"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Prelom v lie\u010dbe f508del - Slan\u00e9 deti<\/title>\n<meta name=\"description\" content=\"V\u00fdsledky klinickej \u0161t\u00fadie preuk\u00e1zali v\u00fdznamn\u00e9 zlep\u0161enia p\u013e\u00facnych funkci\u00ed u naj\u010dastej\u0161ie sa vyskytuj\u00facej mut\u00e1cie f508del.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/slanedeti.sk\/cms\/prelom-v-liecbe-f508del\/1710\" \/>\n<meta property=\"og:locale\" content=\"sk_SK\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Prelom v lie\u010dbe f508del - 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