Christopher J Patriquin, Andrija Bogdanovic, Morag Griffin, Richard J Kelly, Jaroslaw P Maciejewski, Brian Mulherin, Régis Peffault de Latour, Alexander Röth, Veena Selvaratnam, Jeffrey Szer, Mohammed Al-Adhami, Regina Horneff, Lisa Tan, Michael Yeh, Jens Panse
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 safety and efficacy of pegcetacoplan in adult patients with PNH who completed a pegcetacoplan parent study...
April 4, 2024: Advances in Therapy