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PNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threatening anemia, thrombosis, and bone marrow dysfunction. About half of people with the condition die from thrombotic complications.
Ravulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells. However, 10%-20% of patients treated with the antibody infusions experience significant extravascular hemolysis, in which these surviving red blood cells are eliminated by the spleen and liver. Extravascular hemolysis can lead to ongoing anemia, which can lead patients to require blood transfusions.
Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis.
Approval of the oral medication was based on the phase 3 ALPHA trial in 63 patients with PNH who received ravulizumab or eculizumab and experienced significant extravascular hemolysis. These patients were randomized 2:1 to either danicopan or placebo.
Danicopan add-on significantly improved hemoglobin concentrations at 12 weeks (least squares mean improvement from baseline: 2.94 g/dL with danicopan vs 0.50 g/dL with placebo) and made transfusions less likely.
Headache, nausea, arthralgia, and diarrhea were the most common treatment-emergent side effects. Serious adverse events in the danicopan group included cholecystitis and COVID-19 in one patient each.
Danicopan carries a boxed warning of serious infections and is available only through a Risk Evaluation and Mitigation Strategy program.
A version of this article appeared on Medscape.com.
PNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threatening anemia, thrombosis, and bone marrow dysfunction. About half of people with the condition die from thrombotic complications.
Ravulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells. However, 10%-20% of patients treated with the antibody infusions experience significant extravascular hemolysis, in which these surviving red blood cells are eliminated by the spleen and liver. Extravascular hemolysis can lead to ongoing anemia, which can lead patients to require blood transfusions.
Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis.
Approval of the oral medication was based on the phase 3 ALPHA trial in 63 patients with PNH who received ravulizumab or eculizumab and experienced significant extravascular hemolysis. These patients were randomized 2:1 to either danicopan or placebo.
Danicopan add-on significantly improved hemoglobin concentrations at 12 weeks (least squares mean improvement from baseline: 2.94 g/dL with danicopan vs 0.50 g/dL with placebo) and made transfusions less likely.
Headache, nausea, arthralgia, and diarrhea were the most common treatment-emergent side effects. Serious adverse events in the danicopan group included cholecystitis and COVID-19 in one patient each.
Danicopan carries a boxed warning of serious infections and is available only through a Risk Evaluation and Mitigation Strategy program.
A version of this article appeared on Medscape.com.
PNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threatening anemia, thrombosis, and bone marrow dysfunction. About half of people with the condition die from thrombotic complications.
Ravulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells. However, 10%-20% of patients treated with the antibody infusions experience significant extravascular hemolysis, in which these surviving red blood cells are eliminated by the spleen and liver. Extravascular hemolysis can lead to ongoing anemia, which can lead patients to require blood transfusions.
Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis.
Approval of the oral medication was based on the phase 3 ALPHA trial in 63 patients with PNH who received ravulizumab or eculizumab and experienced significant extravascular hemolysis. These patients were randomized 2:1 to either danicopan or placebo.
Danicopan add-on significantly improved hemoglobin concentrations at 12 weeks (least squares mean improvement from baseline: 2.94 g/dL with danicopan vs 0.50 g/dL with placebo) and made transfusions less likely.
Headache, nausea, arthralgia, and diarrhea were the most common treatment-emergent side effects. Serious adverse events in the danicopan group included cholecystitis and COVID-19 in one patient each.
Danicopan carries a boxed warning of serious infections and is available only through a Risk Evaluation and Mitigation Strategy program.
A version of this article appeared on Medscape.com.
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ALEXANDER OTTO, PA, MMS</bylineFull> <bylineTitleText/> <USOrGlobal/> <wireDocType/> <newsDocType/> <journalDocType/> <linkLabel/> <pageRange/> <citation/> <quizID/> <indexIssueDate/> <itemClass qcode="ninat:text"/> <provider qcode="provider:imng"> <name>IMNG Medical Media</name> <rightsInfo> <copyrightHolder> <name>Frontline Medical News</name> </copyrightHolder> <copyrightNotice>Copyright (c) 2015 Frontline Medical News, a Frontline Medical Communications Inc. company. All rights reserved. This material may not be published, broadcast, copied, or otherwise reproduced or distributed without the prior written permission of Frontline Medical Communications Inc.</copyrightNotice> </rightsInfo> </provider> <abstract/> <metaDescription>The US Food and Drug Administration has approved danicopan (Voydeya, AstraZeneca) as an add-on therapy to treat extravascular hemolysis in adults receiving ravu</metaDescription> <articlePDF/> <teaserImage/> <teaser>Danicopan to support treatment of a rare blood disorder, paroxysmal nocturnal hemoglobinuria, is approved by the Food and Drug Administration. </teaser> <title>FDA OKs Danicopan Add-On for Extravascular Hemolysis in Adults With PNH</title> <deck/> <disclaimer/> <AuthorList/> <articleURL/> <doi/> <pubMedID/> <publishXMLStatus/> <publishXMLVersion>1</publishXMLVersion> <useEISSN>0</useEISSN> <urgency/> <pubPubdateYear/> <pubPubdateMonth/> <pubPubdateDay/> <pubVolume/> <pubNumber/> <wireChannels/> <primaryCMSID/> <CMSIDs/> <keywords/> <seeAlsos/> <publications_g> <publicationData> <publicationCode>hemn</publicationCode> <pubIssueName/> <pubArticleType/> <pubTopics/> <pubCategories/> <pubSections/> </publicationData> </publications_g> <publications> <term canonical="true">18</term> </publications> <sections> <term canonical="true">37225</term> <term>39313</term> </sections> <topics> <term canonical="true">191</term> <term>304</term> </topics> <links/> </header> <itemSet> <newsItem> <itemMeta> <itemRole>Main</itemRole> <itemClass>text</itemClass> <title>FDA OKs Danicopan Add-On for Extravascular Hemolysis in Adults With PNH</title> <deck/> </itemMeta> <itemContent> <p> <span class="tag metaDescription">The US Food and Drug Administration has approved danicopan (Voydeya, AstraZeneca) as an add-on therapy to treat extravascular hemolysis in adults receiving ravulizumab or <span class="Hyperlink"><a href="https://reference.medscape.com/drug/soliris-eculizumab-342875">eculizumab</a></span> for paroxysmal nocturnal hemoglobinuria (PNH), according to a <span class="Hyperlink"><a href="https://www.astrazeneca-us.com/media/press-releases/2024/voydeya-approved-in-the-us-as-add-on-therapy-to-ravulizumab-or-eculizumab-for-treatment-of-extravascular-hemolysis-in-adults-with-the-rare-disease-phn.html">press release</a></span> from AstraZeneca.</span> </p> <p>PNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threatening <span class="Hyperlink">anemia</span>, thrombosis, and bone marrow dysfunction. About half of people with the condition <span class="Hyperlink">die from thrombotic complications</span>.<br/><br/>Ravulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells. However, 10%-20% of patients treated with the antibody infusions experience significant extravascular hemolysis, in which these surviving red blood cells are eliminated by the spleen and liver. Extravascular hemolysis can lead to ongoing anemia, which can lead patients to require blood transfusions.<br/><br/>Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis.<br/><br/>Approval of the oral medication was based on the <span class="Hyperlink"><a href="https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(23)00315-0/abstract">phase 3 ALPHA trial</a></span> in 63 patients with PNH who received ravulizumab or eculizumab and experienced significant extravascular hemolysis. These patients were randomized 2:1 to either danicopan or placebo.<br/><br/>Danicopan add-on significantly improved hemoglobin concentrations at 12 weeks (least squares mean improvement from baseline: 2.94 g/dL with danicopan vs 0.50 g/dL with placebo) and made transfusions less likely.<br/><br/><span class="Hyperlink">Headache</span>, nausea, arthralgia, and <span class="Hyperlink">diarrhea</span> were the most common treatment-emergent side effects. Serious adverse events in the danicopan group included <span class="Hyperlink">cholecystitis</span> and COVID-19 in one patient each.<br/><br/>Danicopan carries a <span class="Hyperlink"><a href="https://alexion.com/Documents/voydeya_uspi">boxed warning</a></span> of serious infections and is available only through a Risk Evaluation and Mitigation Strategy program.<span class="end"/></p> <p> <em>A version of this article appeared on <a href="https://www.medscape.com/viewarticle/fda-oks-danicopan-add-extravascular-hemolysis-adults-pnh-2024a100067w?src=">Medscape.com</a>.</em> </p> </itemContent> </newsItem> <newsItem> <itemMeta> <itemRole>teaser</itemRole> <itemClass>text</itemClass> <title/> <deck/> </itemMeta> <itemContent> </itemContent> </newsItem> </itemSet></root>