{"id":49887,"date":"2025-11-04T15:04:21","date_gmt":"2025-11-04T18:04:21","guid":{"rendered":"https:\/\/academiadepacientes.com.br\/?p=49887"},"modified":"2025-11-04T15:04:24","modified_gmt":"2025-11-04T18:04:24","slug":"sarepta-tenta-aprovacao-plena-apos-fracasso-em-estudo-elevidys-perde-indicacao","status":"publish","type":"post","link":"https:\/\/academiadepacientes.com.br\/?p=49887","title":{"rendered":"Sarepta tenta aprova\u00e7\u00e3o plena ap\u00f3s fracasso em estudo; Elevidys perde indica\u00e7\u00e3o"},"content":{"rendered":"\n<p>\ud83e\uddec A Sarepta falhou no estudo confirmat\u00f3rio para Vyondys\/Amondys, mas ainda buscar\u00e1 aprova\u00e7\u00e3o plena apoiada em dados do mundo real. Elevidys ter\u00e1 r\u00f3tulo reduzido e alerta de hepatotoxicidade.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>Por que isso importa:<\/strong> A disputa exp\u00f5e os limites da aprova\u00e7\u00e3o acelerada em distrofia muscular de Duchenne e pressiona a FDA sobre evid\u00eancia cl\u00ednica m\u00ednima para manter terapias no mercado.<\/li>\n<\/ul>\n\n\n\n<p>\ud83e\udde0 <strong>O que est\u00e1 acontecendo:<\/strong><br>O estudo Essence n\u00e3o atingiu signific\u00e2ncia estat\u00edstica no desfecho prim\u00e1rio (0,05 passo\/s; p=0,309). A empresa alega que dados observacionais sustentam benef\u00edcio.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>O estudo p\u00f3s-hoc \u201clivre de COVID\u201d apresentou um valor de p igual a 0,09.<\/li>\n\n\n\n<li>Desfecho prim\u00e1rio foi alterado recentemente.<\/li>\n<\/ul>\n\n\n\n<p>\ud83d\udd0d <strong>Nas Entrelinhas:<\/strong><br>A Sarepta quer converter aprova\u00e7\u00f5es aceleradas para plenas, apesar do fracasso cl\u00ednico formal.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>O argumento da Sarepta se ancora em uma seguran\u00e7a est\u00e1vel e em dados do mundo real.<\/li>\n<\/ul>\n\n\n\n<p>\ud83c\udfc3 <strong>Resumindo:<\/strong><br>Elevidys caiu em vendas no 3\u00ba trimestre e perder\u00e1 indica\u00e7\u00e3o para pacientes n\u00e3o deambulantes (sem capacidade de andar) ap\u00f3s mortes por insufici\u00eancia hep\u00e1tica.<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Empresa testar\u00e1 sirolimo para redu\u00e7\u00e3o de toxicidade.<\/li>\n<\/ul>\n\n\n\n<p>\ud83d\uddbc\ufe0f <strong>O quadro geral:<\/strong><br>FDA, cl\u00ednicos e pagadores precisar\u00e3o decidir se an\u00e1lises p\u00f3s-hoc e evid\u00eancias do mundo real bastam para manter amplo acesso, enquanto riscos hep\u00e1ticos pressionam o r\u00f3tulo.<\/p>\n\n\n\n<p>\ud83d\udcad <strong>Nossa opini\u00e3o:<\/strong><br>A aprova\u00e7\u00e3o plena \u00e9 poss\u00edvel, mas o cen\u00e1rio favorece limita\u00e7\u00f5es adicionais de uso.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p>Fonte: <a href=\"https:\/\/www.fiercepharma.com\/pharma\/sarepta-pushes-full-approval-duchenne-drugs-despite-missed-confirmatory-trial-goal?utm_medium=email&amp;utm_source=nl&amp;utm_campaign=LS-NL-FiercePharma&amp;oly_enc_id=7898C7345667D5R\" target=\"_blank\" rel=\"noopener\" title=\"\">Sarepta pushes for full approval of Duchenne drugs despite missed confirmatory trial goal<\/a> \/ Fierce Pharma<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Sarepta falha em estudo de distrofia muscular de Duchenne, busca aprova\u00e7\u00e3o plena com dados do mundo real; Elevidys perde indica\u00e7\u00e3o por riscos hep\u00e1ticos.<\/p>\n","protected":false},"author":29,"featured_media":48737,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"om_disable_all_campaigns":false,"_uag_custom_page_level_css":"","_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"footnotes":""},"categories":[92],"tags":[],"class_list":["post-49887","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-noticias","infinite-scroll-item","resize-featured-image"],"aioseo_notices":[],"uagb_featured_image_src":{"full":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",1024,1024,false],"thumbnail":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:150\/h:150\/q:mauto\/rt:fill\/g:ce\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",150,150,true],"medium":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:300\/h:300\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",300,300,true],"medium_large":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:768\/h:768\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",768,768,true],"large":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",1024,1024,false],"1536x1536":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",1024,1024,false],"2048x2048":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",1024,1024,false],"mailpoet_newsletter_max":["https:\/\/mlzfzsux7b5d.i.optimole.com\/w:auto\/h:auto\/q:mauto\/f:best\/https:\/\/academiadepacientes.com.br\/wp-content\/uploads\/2024\/06\/tubos-de-ensaio.png",1024,1024,false]},"uagb_author_info":{"display_name":"Equipe Editorial","author_link":"https:\/\/academiadepacientes.com.br\/?author=29"},"uagb_comment_info":0,"uagb_excerpt":"Sarepta falha em estudo de distrofia muscular de Duchenne, busca aprova\u00e7\u00e3o plena com dados do mundo real; Elevidys perde indica\u00e7\u00e3o por riscos hep\u00e1ticos.","_links":{"self":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts\/49887","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/users\/29"}],"replies":[{"embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=49887"}],"version-history":[{"count":1,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts\/49887\/revisions"}],"predecessor-version":[{"id":49888,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/posts\/49887\/revisions\/49888"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=\/wp\/v2\/media\/48737"}],"wp:attachment":[{"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=49887"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=49887"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/academiadepacientes.com.br\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=49887"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}