{"id":5244,"date":"2017-03-14T15:15:10","date_gmt":"2017-03-14T18:15:10","guid":{"rendered":"https:\/\/prodoctor.net\/blog\/?p=5244"},"modified":"2021-06-17T13:43:19","modified_gmt":"2021-06-17T16:43:19","slug":"doencas-raras-ganham-dois-novos-tratamentos","status":"publish","type":"post","link":"https:\/\/prodoctor.net\/blog\/doencas-raras-ganham-dois-novos-tratamentos\/","title":{"rendered":"Doen\u00e7as raras ganham dois novos tratamentos"},"content":{"rendered":"<p>Dois medicamentos in\u00e9ditos para <strong>doen\u00e7as raras ganharam registro da Anvisa<\/strong>. Os novos produtos s\u00e3o o <strong>eculizumabe e o hemitartarato de eliglustate<\/strong>. Os dois registros foram publicados nesta segunda-feira (13\/3) no Di\u00e1rio Oficial da Uni\u00e3o.<\/p>\n<p>O eculizumabe, registrado com o nome comercial de Soliris, <strong>foi aprovado para um tipo de doen\u00e7a que afeta o sistema sangu\u00edneo<\/strong> e \u00e9 denominada <strong>Hemoglobin\u00faria Parox\u00edstica Noturna (HPN)<\/strong>. Nos pacientes com HPN, os gl\u00f3bulos vermelhos <strong>podem ser destru\u00eddos pela a\u00e7\u00e3o do Sistema Complemento<\/strong>, o que leva a valores baixos nas contagens de gl\u00f3bulos vermelhos (anemia), fadiga, dificuldade de funcionamento de diversos \u00f3rg\u00e3os, dores cr\u00f4nicas, urina escura, falta de ar e co\u00e1gulos sangu\u00edneos. O eculizumabe <strong>foi registrado como medicamento biol\u00f3gico novo<\/strong>.<\/p>\n<p>O medicamento tamb\u00e9m ser\u00e1 indicado para o<strong> tratamento de adultos e crian\u00e7as<\/strong> com um tipo de doen\u00e7a que afeta o sistema sangu\u00edneo e os rins denominada <strong>S\u00edndrome Hemol\u00edtico Ur\u00e9mica at\u00edpica (SHUa)<\/strong>. Nestes pacientes podem ocorrer valores baixos nas contagens das c\u00e9lulas sangu\u00edneas (trombocitopenia e anemia), perda ou redu\u00e7\u00e3o da fun\u00e7\u00e3o dos rins, co\u00e1gulos sangu\u00edneos, fadiga e dificuldade de funcionamento de diversos \u00f3rg\u00e3os.<\/p>\n<h3>Como funciona o Soliris (eculizumabe)<\/h3>\n<p>O eculizumabe pode <strong>bloquear a a\u00e7\u00e3o do complemento<\/strong>, a resposta inflamat\u00f3ria do organismo e a sua capacidade de atacar e <strong>destruir as pr\u00f3prias c\u00e9lulas sangu\u00edneas vulner\u00e1veis<\/strong> (c\u00e9lulas HPN).<\/p>\n<p>A evid\u00eancia do benef\u00edcio cl\u00ednico de Soliris\u00ae (eculizumabe) foi demonstrada no <strong>tratamento de pacientes com hem\u00f3lise<\/strong> e sintoma(s) cl\u00ednico(s) indicativo(s) de elevada atividade da doen\u00e7a, independente do hist\u00f3rico de transfus\u00f5es.<\/p>\n<h3>Doen\u00e7a de Gaucher tem novo tratamento<\/h3>\n<p>J\u00e1 o hemitartarato de eliglustate foi aprovado com o<strong> nome comercial de Cerdelga<\/strong>. O produto ser\u00e1 produzido na forma de c\u00e1psulas de 100mg. O medicamento \u00e9 indicado para o <strong>tratamento, de longo prazo<\/strong>, de pacientes adultos com <strong>Doen\u00e7a de Gaucher tipo 1 (DG1)<\/strong>, que s\u00e3o metabolizadores pobres (MP), intermedi\u00e1rios (MI) ou extensos (ME) do <strong>CYP2D6<\/strong>.<\/p>\n<p>A Doen\u00e7a de Gaucher \u00e9 uma <strong>doen\u00e7a gen\u00e9tica, progressiva<\/strong>, e a mais comum das doen\u00e7as lisoss\u00f4micas de dep\u00f3sito, que recebem esse nome devido ao <strong>ac\u00famulo de restos de c\u00e9lulas envelhecidas<\/strong> depositadas nos lisossomos (pequenas estruturas celulares que cont\u00eam enzimas essenciais ao equil\u00edbrio do organismo). Os sintomas mais comuns s\u00e3o cansa\u00e7o, dores nos ossos, fraturas espont\u00e2neas, cirrose, entre outros.<\/p>\n<p>O novo medicamento Cerdelga (hemitartarato de eliglustate), <strong>ser\u00e1 fabricado pela empresa Genzyme<\/strong> localizada em Waterford, Irlanda, e a dona do registro do medicamento no Brasil \u00e9 a empresa<strong> Genzyme do Brasil Ltda<\/strong>., localizada em S\u00e3o Paulo-SP.<\/p>\n<p>Fonte: <a href=\"http:\/\/portal.anvisa.gov.br\/noticias\/-\/asset_publisher\/FXrpx9qY7FbU\/content\/doencas-raras-ganham-dois-novos-tratamentos\/219201?p_p_auth=fmOapOgU&amp;inheritRedirect=false&amp;redirect=http%3A%2F%2Fportal.anvisa.gov.br%2Fnoticias%3Fp_p_auth%3DfmOapOgU%26p_p_id%3D101_INSTANCE_FXrpx9qY7FbU%26p_p_lifecycle%3D0%26p_p_state%3Dnormal%26p_p_mode%3Dview%26p_p_col_id%3D_118_INSTANCE_dKu0997DQuKh__column-2%26p_p_col_count%3D2\" target=\"_blank\" rel=\"noopener\">Anvisa<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Dois medicamentos in\u00e9ditos para doen\u00e7as raras ganharam registro da Anvisa. Os novos produtos s\u00e3o o eculizumabe e o hemitartarato&#8230;<\/p>\n","protected":false},"author":1,"featured_media":19814,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_kad_blocks_custom_css":"","_kad_blocks_head_custom_js":"","_kad_blocks_body_custom_js":"","_kad_blocks_footer_custom_js":"","_kadence_starter_templates_imported_post":false,"_kad_post_transparent":"","_kad_post_title":"","_kad_post_layout":"","_kad_post_sidebar_id":"","_kad_post_content_style":"","_kad_post_vertical_padding":"","_kad_post_feature":"","_kad_post_feature_position":"","_kad_post_header":false,"_kad_post_footer":false,"footnotes":""},"categories":[415],"tags":[],"class_list":["post-5244","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-medicamentos-e-farmacia"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ 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