{"id":4357,"date":"2025-08-20T17:19:30","date_gmt":"2025-08-20T14:19:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4357"},"modified":"2025-08-20T17:21:43","modified_gmt":"2025-08-20T14:21:43","slug":"keros-announces-u-s-fda-orphan-drug-designation-granted-to-ker-065-for-the-treatment-of-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/keros-announces-u-s-fda-orphan-drug-designation-granted-to-ker-065-for-the-treatment-of-duchenne-muscular-dystrophy\/","title":{"rendered":"Keros anuncia a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o dos EUA para o FDA concedida ao KER-065 para o tratamento da distrofia muscular de Duchenne"},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Keros Therapeutics anunciou hoje que a Food and Drug Administration (\u201cFDA\u201d) concedeu a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o para o KER-065 para o tratamento da distrofia muscular de Duchenne (\u201cDMD\u201d).<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201cReceber a designa\u00e7\u00e3o de Medicamento \u00d3rf\u00e3o para o KER-065 destaca a significativa necessidade m\u00e9dica n\u00e3o atendida de pacientes com DMD\u201d, disse Jasbir S. Seehra, Presidente e CEO. \u201cEsta designa\u00e7\u00e3o representa um marco significativo para o Keros, \u00e0 medida que avan\u00e7amos com o KER-065 para um ensaio cl\u00ednico de Fase 2 em pacientes com DMD.\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O FDA concede a designa\u00e7\u00e3o de Medicamento \u00d3rf\u00e3o a terapias experimentais que abordam doen\u00e7as ou condi\u00e7\u00f5es m\u00e9dicas raras que afetam menos de 200.000 pessoas nos Estados Unidos. Essa designa\u00e7\u00e3o oferece certos benef\u00edcios potenciais, incluindo cr\u00e9ditos fiscais para testes cl\u00ednicos qualificados, isen\u00e7\u00e3o ou pagamento parcial das taxas de solicita\u00e7\u00e3o do FDA e sete anos de exclusividade de mercado, se aprovado.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ler mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/keros-therapeutics-announces-initial-topline-results-from-the-phase-1-clinical-trial-of-ker-065-in-healthy-volunteers\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ensaio cl\u00ednico de fase 1 do KER-065 em volunt\u00e1rios saud\u00e1veis<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">O que \u00e9 KER-065?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">KER-065 \u00e9 uma nova armadilha de ligante composta por um dom\u00ednio de liga\u00e7\u00e3o de ligante modificado derivado do receptor de activina tipo IIA e do receptor de activina tipo IIB que \u00e9 fundido \u00e0 por\u00e7\u00e3o do anticorpo humano conhecida como dom\u00ednio Fc. KER-065 foi desenvolvido para atuar como uma armadilha de ligante e inibir os efeitos biol\u00f3gicos da miostatina e da activina A, dois ligantes que sinalizam por meio de receptores de activina, para aumentar a regenera\u00e7\u00e3o do m\u00fasculo esquel\u00e9tico, aumentar o tamanho e a for\u00e7a muscular, reduzir a gordura corporal, reduzir a fibrose do m\u00fasculo esquel\u00e9tico e aumentar a resist\u00eancia \u00f3ssea.<\/p>","protected":false},"excerpt":{"rendered":"<p>Keros Therapeutics today announced the U.S. Food and Drug Administration (\u201cFDA\u201d) granted Orphan Drug designation for KER-065 for the treatment of Duchenne muscular dystrophy (\u201cDMD\u201d). \u201cReceiving Orphan Drug designation for KER-065 highlights the significant unmet medical need for patients with DMD,\u201d said Jasbir S. Seehra, President and Chief Executive Officer. \u201cThis designation serves as a [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4360,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[337,336,195],"class_list":["post-4357","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-ker-065","tag-keros-therapeutics","tag-orphan-drug"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4357","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=4357"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4357\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4360"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4357"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4357"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4357"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}