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

ConclusionPegcetacoplan sustained long-term improvements in hemoglobin concentrations, fatigue reduction, and transfusion burden. Long-term safety findings corroborate the favorable profile established for pegcetacoplan.Trial RegistrationClinicalTrials.gov identifier, NCT03531255.

http://link.springer.com/10.1007/s12325-024-02827-8

Source: Advances in Therapy - April 4, 2024 Category: Drugs & Pharmacology Source Type: research