Summary of Clinical Use

Pegcetacoplan was granted orphan medicine/drug designations by the EMA (2017) and FDA (2014), to treat paroxysmal nocturnal haemoglobinuria (PNH) prior to full marketing approvals for this indication by both agencies in 2021 [3,7]. The FDA has also issued orphan drug designations for pegcetacoplan to be used to treat additional rare complement-mediated conditions including C3 glomerulopathy (2018), autoimmune hemolytic anemia (2019) [1] and immune complex-membranoproliferative glomerulonephritis (2022), as well as amyotrophic lateral sclerosis (2023).

Clinical Trials
Clinical Trial ID	Title	Type	Source	Comment	References
NCT04901936	A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 2 Interventional	Apellis Pharmaceuticals, Inc.
NCT05067127	Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.
NCT05096403	A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)	Phase 3 Interventional	Swedish Orphan Biovitrum
NCT05809531	An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.
NCT03531255	Pegcetacoplan Long Term Safety and Efficacy Extension Study	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.
NCT04085601	A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.
NCT04770545	An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.
NCT03500549	Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.		8
NCT03525613	A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration	Phase 3 Interventional	Apellis Pharmaceuticals, Inc.		2

External links

For extended ADME data see the following: Drugs.com European Medicines Agency (EMA)