User login
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use (CHMP).
PNH is a rare, debilitating, and potentially life-threatening genetic disorder that causes hemolytic anemia. Symptoms of the disease include fatigue, body pain, blood clots, bleeding, and shortness of breath. The standard treatment is anti-C5 monoclonal antibodies (eculizumab or ravulizumab) via subcutaneous or intravenous infusion. However, a minority of patients with PNH who are treated with these complement inhibitors encounter residual hemolytic anemia and require red blood cell transfusions.
The active substance of Fabhalta is iptacopan, a proximal complement inhibitor. Iptacopan targets factor B to selectively inhibit the alternative complement pathway and control both C3-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.
Superior Results in Phase 3 Trials
The decision to grant a marketing authorization was taken following a review of two phase 3 trials. The main study was a randomized, open-label, active comparator trial involving 97 patients with PNH who had residual anemia despite receiving treatment with anti-C5 monoclonal antibodies for the previous 6 months. Of the trial participants, 62 received iptacopan monotherapy and 35 continued their anti-C5 regimen for 24 weeks.
Treatment with Fabhalta was found to be significantly superior to the anti-C5 regimen, with 51 of 60 patients who could be evaluated achieving hemoglobin improvement (≥ 2 g/dL) and 42 achieving sustained hemoglobin levels (≥ 12 g/dL) without transfusion, compared with no patients who continued treatment with anti-C5 monoclonal antibodies. Also, 59 of 62 patients treated with Fabhalta did not require blood transfusions between day 14 and day 168, compared with 14 of 35 patients in the anti-C5 group.
The second trial was a single-arm study involving 40 PNH patients who had not previously been treated with a complement inhibitor. Following treatment with Fabhalta, 31 of 33 patients who could be evaluated achieved hemoglobin improvement (≥ 2 g/dL) at week 24, whereas 19 achieved sustained hemoglobin levels (≥ 12 g/dL) without transfusion.
The most common side effects of Fabhalta are upper respiratory tract infection, headache, and diarrhea.
The CHMP stressed that Fabhalta should be prescribed by physicians who are experienced in the management of patients with hematologic disorders.
Fabhalta was supported through the EMA’s Priority Medicines (PRIME) scheme, which provides regulatory support for promising medicines with the potential to address unmet medical needs. The CHMP’s recommendation has been sent to the European Commission for a final decision.
Novartis said in a company statement on March 22 that, if approved, Fabhalta would be the first oral monotherapy available to PNH patients in Europe.
A version of this article appeared on Medscape.com.
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use (CHMP).
PNH is a rare, debilitating, and potentially life-threatening genetic disorder that causes hemolytic anemia. Symptoms of the disease include fatigue, body pain, blood clots, bleeding, and shortness of breath. The standard treatment is anti-C5 monoclonal antibodies (eculizumab or ravulizumab) via subcutaneous or intravenous infusion. However, a minority of patients with PNH who are treated with these complement inhibitors encounter residual hemolytic anemia and require red blood cell transfusions.
The active substance of Fabhalta is iptacopan, a proximal complement inhibitor. Iptacopan targets factor B to selectively inhibit the alternative complement pathway and control both C3-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.
Superior Results in Phase 3 Trials
The decision to grant a marketing authorization was taken following a review of two phase 3 trials. The main study was a randomized, open-label, active comparator trial involving 97 patients with PNH who had residual anemia despite receiving treatment with anti-C5 monoclonal antibodies for the previous 6 months. Of the trial participants, 62 received iptacopan monotherapy and 35 continued their anti-C5 regimen for 24 weeks.
Treatment with Fabhalta was found to be significantly superior to the anti-C5 regimen, with 51 of 60 patients who could be evaluated achieving hemoglobin improvement (≥ 2 g/dL) and 42 achieving sustained hemoglobin levels (≥ 12 g/dL) without transfusion, compared with no patients who continued treatment with anti-C5 monoclonal antibodies. Also, 59 of 62 patients treated with Fabhalta did not require blood transfusions between day 14 and day 168, compared with 14 of 35 patients in the anti-C5 group.
The second trial was a single-arm study involving 40 PNH patients who had not previously been treated with a complement inhibitor. Following treatment with Fabhalta, 31 of 33 patients who could be evaluated achieved hemoglobin improvement (≥ 2 g/dL) at week 24, whereas 19 achieved sustained hemoglobin levels (≥ 12 g/dL) without transfusion.
The most common side effects of Fabhalta are upper respiratory tract infection, headache, and diarrhea.
The CHMP stressed that Fabhalta should be prescribed by physicians who are experienced in the management of patients with hematologic disorders.
Fabhalta was supported through the EMA’s Priority Medicines (PRIME) scheme, which provides regulatory support for promising medicines with the potential to address unmet medical needs. The CHMP’s recommendation has been sent to the European Commission for a final decision.
Novartis said in a company statement on March 22 that, if approved, Fabhalta would be the first oral monotherapy available to PNH patients in Europe.
A version of this article appeared on Medscape.com.
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use (CHMP).
PNH is a rare, debilitating, and potentially life-threatening genetic disorder that causes hemolytic anemia. Symptoms of the disease include fatigue, body pain, blood clots, bleeding, and shortness of breath. The standard treatment is anti-C5 monoclonal antibodies (eculizumab or ravulizumab) via subcutaneous or intravenous infusion. However, a minority of patients with PNH who are treated with these complement inhibitors encounter residual hemolytic anemia and require red blood cell transfusions.
The active substance of Fabhalta is iptacopan, a proximal complement inhibitor. Iptacopan targets factor B to selectively inhibit the alternative complement pathway and control both C3-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.
Superior Results in Phase 3 Trials
The decision to grant a marketing authorization was taken following a review of two phase 3 trials. The main study was a randomized, open-label, active comparator trial involving 97 patients with PNH who had residual anemia despite receiving treatment with anti-C5 monoclonal antibodies for the previous 6 months. Of the trial participants, 62 received iptacopan monotherapy and 35 continued their anti-C5 regimen for 24 weeks.
Treatment with Fabhalta was found to be significantly superior to the anti-C5 regimen, with 51 of 60 patients who could be evaluated achieving hemoglobin improvement (≥ 2 g/dL) and 42 achieving sustained hemoglobin levels (≥ 12 g/dL) without transfusion, compared with no patients who continued treatment with anti-C5 monoclonal antibodies. Also, 59 of 62 patients treated with Fabhalta did not require blood transfusions between day 14 and day 168, compared with 14 of 35 patients in the anti-C5 group.
The second trial was a single-arm study involving 40 PNH patients who had not previously been treated with a complement inhibitor. Following treatment with Fabhalta, 31 of 33 patients who could be evaluated achieved hemoglobin improvement (≥ 2 g/dL) at week 24, whereas 19 achieved sustained hemoglobin levels (≥ 12 g/dL) without transfusion.
The most common side effects of Fabhalta are upper respiratory tract infection, headache, and diarrhea.
The CHMP stressed that Fabhalta should be prescribed by physicians who are experienced in the management of patients with hematologic disorders.
Fabhalta was supported through the EMA’s Priority Medicines (PRIME) scheme, which provides regulatory support for promising medicines with the potential to address unmet medical needs. The CHMP’s recommendation has been sent to the European Commission for a final decision.
Novartis said in a company statement on March 22 that, if approved, Fabhalta would be the first oral monotherapy available to PNH patients in Europe.
A version of this article appeared on Medscape.com.
<!--$RCSfile: InCopy_agile.xsl,v $ $Revision: 1.35 $-->
<!--$RCSfile: drupal.xsl,v $ $Revision: 1.7 $-->
<root generator="drupal.xsl" gversion="1.7"> <header> <fileName>167432</fileName> <TBEID>0C04F371.SIG</TBEID> <TBUniqueIdentifier>MD_0C04F371</TBUniqueIdentifier> <newsOrJournal>News</newsOrJournal> <publisherName>Frontline Medical Communications</publisherName> <storyname/> <articleType>2</articleType> <TBLocation>QC Done-All Pubs</TBLocation> <QCDate>20240325T100800</QCDate> <firstPublished>20240325T101548</firstPublished> <LastPublished>20240325T101548</LastPublished> <pubStatus qcode="stat:"/> <embargoDate/> <killDate/> <CMSDate>20240325T101548</CMSDate> <articleSource/> <facebookInfo/> <meetingNumber/> <byline>Peter Russell</byline> <bylineText>PETER RUSSELL</bylineText> <bylineFull>PETER RUSSELL</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 European Medicines Agency (EMA) has granted a marketing authorization to Novartis Europharm for Fabhalta (iptacopan) for treating adults with paroxysmal noc</metaDescription> <articlePDF/> <teaserImage/> <teaser>The European Medicines Agency has greenlighted the sale of Fabhalta for adult patients with a rare bleeding disorder. </teaser> <title>EU Backs First Oral Monotherapy for 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> </topics> <links/> </header> <itemSet> <newsItem> <itemMeta> <itemRole>Main</itemRole> <itemClass>text</itemClass> <title>EU Backs First Oral Monotherapy for Adults With PNH</title> <deck/> </itemMeta> <itemContent> <p><br/><br/><span class="tag metaDescription">The European Medicines Agency (EMA) has granted a <span class="Hyperlink"><a href="https://www.ema.europa.eu/en/news/first-oral-monotherapy-patients-paroxysmal-nocturnal-haemoglobinuria">marketing authorization</a></span> to Novartis Europharm for <span class="Hyperlink"><a href="https://www.ema.europa.eu/en/medicines/human/EPAR/fabhalta">Fabhalta</a></span> (iptacopan) for treating adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.</span><br/><br/>The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use (CHMP).<br/><br/>PNH is a rare, debilitating, and potentially life-threatening genetic disorder that causes <span class="Hyperlink">hemolytic anemia</span>. Symptoms of the disease include fatigue, body pain, blood clots, bleeding, and shortness of breath. The standard treatment is anti-C5 monoclonal antibodies (<span class="Hyperlink">eculizumab</span> or <span class="Hyperlink">ravulizumab</span>) via subcutaneous or intravenous infusion. However, a minority of patients with PNH who are treated with these complement inhibitors encounter residual hemolytic anemia and require red blood cell transfusions.<br/><br/>The active substance of Fabhalta is iptacopan, a proximal complement inhibitor. Iptacopan targets factor B to selectively inhibit the alternative complement pathway and control both C3-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.</p> <h2>Superior Results in Phase 3 Trials</h2> <p>The decision to grant a marketing authorization was taken following a review of two phase 3 trials. The main study was a randomized, open-label, active comparator trial involving 97 patients with PNH who had residual anemia despite receiving treatment with anti-C5 monoclonal antibodies for the previous 6 months. Of the trial participants, 62 received iptacopan monotherapy and 35 continued their anti-C5 regimen for 24 weeks.<br/><br/>Treatment with Fabhalta was found to be significantly superior to the anti-C5 regimen, with 51 of 60 patients who could be evaluated achieving hemoglobin improvement (≥ 2 g/dL) and 42 achieving sustained hemoglobin levels (≥ 12 g/dL) without transfusion, compared with no patients who continued treatment with anti-C5 monoclonal antibodies. Also, 59 of 62 patients treated with Fabhalta did not require blood transfusions between day 14 and day 168, compared with 14 of 35 patients in the anti-C5 group.<br/><br/>The second trial was a single-arm study involving 40 PNH patients who had not previously been treated with a complement inhibitor. Following treatment with Fabhalta, 31 of 33 patients who could be evaluated achieved hemoglobin improvement (≥ 2 g/dL) at week 24, whereas 19 achieved sustained hemoglobin levels (≥ 12 g/dL) without transfusion.<br/><br/>The most common side effects of Fabhalta are upper respiratory tract infection, <span class="Hyperlink">headache</span>, and <span class="Hyperlink">diarrhea</span>.<br/><br/>The CHMP stressed that Fabhalta should be prescribed by physicians who are experienced in the management of patients with hematologic disorders.<br/><br/>Fabhalta was supported through the EMA’s Priority Medicines (PRIME) scheme, which provides regulatory support for promising medicines with the potential to address unmet medical needs. The CHMP’s recommendation has been sent to the European Commission for a final decision.<br/><br/>Novartis said in a <span class="Hyperlink"><a href="https://www.novartis.com/news/media-releases/novartis-fabhalta-iptacopan-receives-positive-chmp-opinion-first-oral-monotherapy-adult-patients-paroxysmal-nocturnal-hemoglobinuria-pnh">company statement</a></span> on March 22 that, if approved, Fabhalta would be the first oral monotherapy available to PNH patients in Europe.</p> <p> <em>A version of this article appeared on <span class="Hyperlink"><a href="https://www.medscape.com/viewarticle/eu-backs-first-oral-monotherapy-adults-pnh-2024a10005ft">Medscape.com</a></span>.</em> </p> </itemContent> </newsItem> <newsItem> <itemMeta> <itemRole>teaser</itemRole> <itemClass>text</itemClass> <title/> <deck/> </itemMeta> <itemContent> </itemContent> </newsItem> </itemSet></root>