{"id":4450,"date":"2021-12-22T09:43:43","date_gmt":"2021-12-22T08:43:43","guid":{"rendered":"https:\/\/aa-pnh.org\/admission-of-pegcetacoplan-for-pnh-patients\/"},"modified":"2022-03-07T11:32:23","modified_gmt":"2022-03-07T10:32:23","slug":"admission-of-pegcetacoplan-for-pnh-patients","status":"publish","type":"post","link":"https:\/\/aa-pnh.org\/en\/admission-of-pegcetacoplan-for-pnh-patients\/","title":{"rendered":"Approval of pegcetacoplan for PNH patients"},"content":{"rendered":"<p><img decoding=\"async\" src=\"https:\/\/aa-pnh.org\/wp-content\/uploads\/2017\/05\/erkrankungen_title.jpg\" alt=\"blood cells\"\/><\/p>\n<p>On 13 December 2021 a <strong>new drug<\/strong> for the treatment of adult patients with PNH was <strong>approved in the EU<\/strong>: The substance <strong>pegcetacoplan<\/strong> which inhibits specific processes in the <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;immune system&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Defence system; system that enables the body to fight off infections and to distinguish between its own and foreign tissues. In humans, the immune system consists of specialised proteins (antibodies), immune cells (white blood cells) and immune organs. It is responsible for our body&amp;#039;s defences(...)&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/immune-system\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">immune system<\/a> will probably be available in Germany in the beginning of 2022. Its trade name is Aspaveli&reg;. <\/p>\n<p><\/p><p>The drug is a so-called <strong>C3 complement inhibitor<\/strong>: The <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;complement system&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;A group of plasma proteins for immune defence against microorganisms&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/complement-system\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">complement system<\/a> is part of the body&rsquo;s own <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;immune system&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Defence system; system that enables the body to fight off infections and to distinguish between its own and foreign tissues. In humans, the immune system consists of specialised proteins (antibodies), immune cells (white blood cells) and immune organs. It is responsible for our body&amp;#039;s defences(...)&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/immune-system\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">immune system<\/a> which in PNH attacks the red blood cells (<a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;erythrocyte&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Red blood cell; erythrocytes make up the majority of cellular blood components. They are formed in the bone marrow, contain haemoglobin (the red pigment in blood ) and have a life expectancy of about 120 days in healthy people. The haemoglobin content of the blood is an important measurement(...)&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/erythrocyte\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">erythrocyte<\/a>s) and destroys them. Pegcetacoplan inhibits a certain part of the <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;complement system&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;A group of plasma proteins for immune defence against microorganisms&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/complement-system\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">complement system<\/a> and thereby the destruction of red blood cells outside the blood vessels (<a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;extravascular&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Outside the blood vessels&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/extravascular\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">extravascular<\/a>), leading to an improved life cycle of the <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;erythrocyte&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Red blood cell; erythrocytes make up the majority of cellular blood components. They are formed in the bone marrow, contain haemoglobin (the red pigment in blood ) and have a life expectancy of about 120 days in healthy people. The haemoglobin content of the blood is an important measurement(...)&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/erythrocyte\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">erythrocyte<\/a>s and improved <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;haemoglobin&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Hb; red blood cell pigment consisting of haem (an iron-containing component) and globin (a protein). Binds, transports and releases oxygen and carbon dioxide. Reference values: women 12-16 g\/dl or 7.5-9.9 mmol\/l, men 14-18 g\/dl or 8.7-11.2 mmol\/l &lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/haemoglobin\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">haemoglobin<\/a> levels.<\/p>\n<p>Pegecetacoplan is approved for patients who remain anaemic despite treatment with a C5 inhibitor (eculizumab or ravulizumab) for at least 3 months. C5 is also part of the <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;complement system&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;A group of plasma proteins for immune defence against microorganisms&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/complement-system\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">complement system<\/a>. The drug is administered twice a week as a subcutaneous <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;infusion&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Injection of a liquid into a vein&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/infusion\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">infusion<\/a>. Among the most common side effects are reactions at the injection site, infections of the upper respiratory tract, headache, abdominal pain, and diarrhoea.<\/p>\n<p><a href=\"https:\/\/www.ema.europa.eu\/documents\/product-information\/aspaveli-epar-product-information_en.pdf\" rel=\"noopener\" target=\"_blank\">&ldquo;<strong>Summary of product characteristics<\/strong>&ldquo;<\/a> by the European Medicines Agency <a href=\"https:\/\/www.ema.europa.eu\/en\" rel=\"noopener\" target=\"_blank\">EMA<\/a>.<\/p>\n<p>The drug is not entirely new: On 5 April of this year, we already reported in detail on the mechanism of action and the current studies on it in a <a href=\"https:\/\/aa-pnh.org\/en\/results-of-the-pegasus-study-on-apl-2\/\">blog post<\/a> on our website.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>On 13 December 2021 a new drug for the treatment of adult patients with PNH was approved in the EU: The substance pegcetacoplan which inhibits specific processes in the <a class=\"glossaryLink\" aria-describedby=\"tt\" data-cmtooltip=\"&lt;div class=glossaryItemTitle&gt;immune system&lt;\/div&gt;&lt;div class=glossaryItemBody&gt;Defence system; system that enables the body to fight off infections and to distinguish between its own and foreign tissues. In humans, the immune system consists of specialised proteins (antibodies), immune cells (white blood cells) and immune organs. It is responsible for our body&amp;#039;s defences(...)&lt;\/div&gt;\" href=\"https:\/\/aa-pnh.org\/en\/glossar\/immune-system\/\" data-gt-translate-attributes='[{\"attribute\":\"data-cmtooltip\", \"format\":\"html\"}]' tabindex=\"0\" role=\"link\">immune system<\/a> will probably be available in Germany in the beginning of 2022. Its trade name&hellip;<\/p>\n","protected":false},"author":7,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"gallery","meta":{"footnotes":""},"categories":[82,86,81,105,80],"tags":[84,85,83],"class_list":["post-4450","post","type-post","status-publish","format-gallery","hentry","category-clinical-studies","category-illness","category-medication","category-pnh-en","category-studies","tag-medication","tag-pnh-en","tag-studies","post_format-post-format-gallery"],"_links":{"self":[{"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/posts\/4450","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/users\/7"}],"replies":[{"embeddable":true,"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/comments?post=4450"}],"version-history":[{"count":0,"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/posts\/4450\/revisions"}],"wp:attachment":[{"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/media?parent=4450"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/categories?post=4450"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/aa-pnh.org\/en\/wp-json\/wp\/v2\/tags?post=4450"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}