Edwin Wong, Carla Nester, Teresa Cavero, Alexandre Karras, Moglie Le Quintrec, Liz Lightstone, Ute Eisenberger, Maria Jose Soler, David Kavanagh, Erica Daina, Manuel Praga, Nicholas R Medjeral-Thomas, Anja Gäckler, Clara Garcia-Carro, Andrea Biondani, Frederique Chaperon, Kenneth Kulmatycki, Julie Milojevic, Nicholas J A Webb, Prasanna Kumar Nidamarthy, Guido Junge, Giuseppe Remuzzi
INTRODUCTION: Complement 3 glomerulopathy (C3G) is a rare inflammatory kidney disease mediated by dysregulation of the alternative complement pathway. No targeted therapy exists for this aggressive glomerulonephritis. Efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) (measured by complement biomarkers) of iptacopan were assessed in patients with C3G. METHODS: In this phase 2, multicenter, open-label, single-arm, nonrandomized study, adults with biopsy-proven, native kidney C3G (native cohort) and kidney transplant recipients with C3G recurrence (recurrent kidney transplant [KT] cohort) received iptacopan twice daily (bid) for 84 days (days 1-21: 10-100 mg; days 22-84: 200 mg)...
December 2023: KI Reports