{"id":3929,"date":"2025-06-17T16:48:33","date_gmt":"2025-06-17T13:48:33","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3929"},"modified":"2025-06-17T16:48:34","modified_gmt":"2025-06-17T13:48:34","slug":"capricor-therapeutics-announces-bmd-orphan-drug-designation-and-dmd-program-regulatory-development-for-deramiocel-cap-1002","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/capricor-therapeutics-announces-bmd-orphan-drug-designation-and-dmd-program-regulatory-development-for-deramiocel-cap-1002\/","title":{"rendered":"Capricor Therapeutics anuncia a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o BMD e o desenvolvimento regulat\u00f3rio do programa DMD para Deramiocel (CAP-1002)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Capricor Therapeutics, empresa de biotecnologia que desenvolve terapias baseadas em c\u00e9lulas transformadoras e exossomos para o tratamento de doen\u00e7as raras, anunciou hoje que a Food and Drug Administration (FDA) dos EUA concedeu a Designa\u00e7\u00e3o de Medicamento \u00d3rf\u00e3o ao Deramiocel (CAP-1002), principal candidato \u00e0 terapia celular da empresa, para o potencial tratamento da Distrofia Muscular de Becker (DMB). Esta designa\u00e7\u00e3o fortalece a posi\u00e7\u00e3o estrat\u00e9gica da Capricor, \u00e0 medida que avan\u00e7a com uma plataforma totalmente integrada voltada para as complica\u00e7\u00f5es card\u00edacas e esquel\u00e9ticas da distrofia muscular e expande o potencial comercial de seu principal ativo. <a href=\"https:\/\/dmdwarrior.com\/pt\/what-is-deramiocel\/\">O que \u00e9 Deramiocel? O que o CAP-1002 faz?<\/a> \u2013<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Capricor Therapeutics prossegue com passos confiantes<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>O projeto US FDA concede a designa\u00e7\u00e3o de medicamento \u00f3rf\u00e3o ao Deramiocel para o tratamento da distrofia muscular de Becker, ampliando o foco do Capricor em doen\u00e7as neuromusculares<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>O Capricor permanece no caminho certo para a data de 31 de agosto de 2025, conforme a Lei de Taxas para Usu\u00e1rios de Medicamentos Prescritos (PDUFA) para Deramiocel na Distrofia Muscular de Duchenne ap\u00f3s a inspe\u00e7\u00e3o pr\u00e9-licen\u00e7a bem-sucedida do FDA<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">A distrofia muscular de Becker, assim como a distrofia muscular de Duchenne (DMD), \u00e9 uma doen\u00e7a neuromuscular progressiva ligada ao cromossomo X que resulta em deteriora\u00e7\u00e3o significativa dos m\u00fasculos esquel\u00e9ticos e card\u00edacos ao longo do tempo. O Deramiocel est\u00e1 sendo desenvolvido para abordar ambos os aspectos da patologia da doen\u00e7a, incluindo a cardiomiopatia, um dos principais contribuintes para a morbidade e mortalidade em pacientes com DMB e DMD.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>O Pedido de Licen\u00e7a Biol\u00f3gica (BLA) do Capricor para Deramiocel em DMD continua sob revis\u00e3o priorit\u00e1ria, com uma data de a\u00e7\u00e3o alvo do PDUFA de 31 de agosto de 2025.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Saber mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/capricor-therapeutics-deramiocel-mid-cycle-review-meeting-with-fda\/\">Capricor Therapeutics anuncia conclus\u00e3o da reuni\u00e3o de revis\u00e3o<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel (CAP-1002), the company\u2019s lead cell therapy candidate, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation strengthens Capricor\u2019s strategic [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3931,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[361,90,86,87,89,85,117,362,88],"class_list":["post-3929","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-bmd","tag-cap-1002","tag-capricor","tag-capricor-therapeutics","tag-cardiomyopathy","tag-deramiocel","tag-dmd","tag-dmd-cardiomyopathy","tag-fda"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/3929","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=3929"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/3929\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/3931"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=3929"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=3929"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=3929"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}