Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48 Weeks: 307 Open-Label Extension Study

  • Published on 07/26/2024
  •  Reading time: 6 min.

Patriquin Christopher J. 1, Bogdanovic Andrija 2, Griffin Morag 3, Kelly Richard J. 3, Maciejewski Jaroslaw P. 4, Mulherin Brian 5,6, Peffault de Latour Régis 7,8, Röth Alexander 9, Selvaratnam Veena 10, Szer Jeffrey 11, Al-Adhami Mohammed 12, Horneff Regina 13, Tan Lisa 13,14, Yeh Michael 12, Panse Jens 15,16

1 Hematology & Apheresis Medicine University Health Network, Toronto General Hospital Toronto ON Canada
2 https://ror.org/02qsmb048 Clinic of Hematology, Clinical Center of Serbia, Faculty of Medicine University of Belgrade Belgrade Serbia
3 https://ror.org/013s89d74 Department of Haematology St. James’s University Hospital Leeds UK
4 https://ror.org/03xjacd83 Department of Translational Hematology and Oncology Research Taussig Cancer Institute, Cleveland Clinic Cleveland OH USA
5 Hematology Oncology of Indiana Indianapolis IN USA
6 Ascension St. Vincent Carmel Carmel IN USA
7 French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria Paris France
8 Assistance Publique–Hôpitaux de Paris, Saint-Louis Hospital Université Paris Cité Paris France
9 Department of Hematology and Stem Cell Transplantation, West German Cancer Center University Hospital Essen Essen Germany
10 Department of Hematology Ampang Hospital Ampang Malaysia
11 https://ror.org/005bvs909 Department of Clinical Haematology The Royal Melbourne Hospital and Peter MacCallum Cancer Centre Melbourne VIC Australia
12 https://ror.org/0231n7e68 Apellis Pharmaceuticals Waltham MA USA
13 https://ror.org/04asvbz37 Swedish Orphan Biovitrum AB Stockholm Sweden
14 Lisa Tan Pharma Consulting Ltd Cambridge UK
15 Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD) Aachen Germany
16 https://ror.org/04xfq0f34 Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation University Hospital RWTH Aachen Aachen Germany

Abstract

Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening disease characterized by complement-mediated hemolysis and thrombosis. Pegcetacoplan, the first targeted complement component 3 (C3) PNH therapy, was safe and efficacious in treatment-naive and pre-treated patients with PNH in five clinical trials.
Methods The 307 open-label extension (OLE) study (NCT03531255) is a non-randomized, multicenter extension study of long-term...

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