Pegcetacoplan

Overview

Pegcetacoplan is a complement inhibitor indicated in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Prior to its FDA approval, patients with PNH were typically treated with the C5 inhibiting monoclonal antibody eculizumab. Patients given eculizumab experienced less hemolysis caused by the membrane attack complex, but were still somewhat susceptible to hemolysis caused by C3b opsonization. Pegcetacoplan was developed out of a need for an inhibitor of complement mediated hemolysis further upstream of C5. Pegcetacoplan is a pegylated C3 inhibitor that can disrupt the processes leading to both forms of hemolysis that threaten patients with PNH. Pegcetacoplan for subcutaneous use was granted FDA approval on 14 May 2021. In February 2023, pegcetacoplan for intravitreal use was approved by the FDA for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.

Indication

Pegcetacoplan is indicated to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also indicated to treat geographic atrophy (GA) secondary to age-related macular degeneration.

Associated Conditions

Geographic Atrophy Secondary to Age-related Macular Degeneration
Paroxysmal Nocturnal Haemoglobinuria (PNH)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
A Phase 3 Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation	2025/06/13	NCT07020832	Phase 3	Not yet recruiting	Apellis Pharmaceuticals, Inc.
A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy	2024/12/09	NCT06722157	Phase 2	Recruiting	Boehringer Ingelheim
A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration	2023/12/08	NCT06161584	N/A	Recruiting	Apellis Pharmaceuticals, Inc.
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	2023/04/12	NCT05809531	Phase 3	Active, not recruiting	Apellis Pharmaceuticals, Inc.
A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	2023/03/20	NCT05776472	N/A	Recruiting	Swedish Orphan Biovitrum
Evaluate the PK Efficacy Safety and Tolerability of Pegcetacoplan in Patients With Thrombotic Microangiopathy	2021/12/08	NCT05148299	Phase 2	Completed	Swedish Orphan Biovitrum
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)	2021/10/27	NCT05096403	Phase 3	Suspended	Swedish Orphan Biovitrum
Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis	2021/10/05	NCT05067127	Phase 3	Completed	Apellis Pharmaceuticals, Inc.
APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion	2021/06/09	NCT04919629	Phase 2	Recruiting	Roswell Park Cancer Institute
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	2021/05/26	NCT04901936	Phase 2	Recruiting	Apellis Pharmaceuticals, Inc.

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Empaveli	Apellis Pharmaceuticals, Inc.	73606-010	SUBCUTANEOUS	1080 mg in 20 mL	7/30/2025
Syfovre	Apellis Pharmaceuticals, Inc.	73606-020	INTRAVITREAL	15 mg in 0.1 mL	7/29/2025

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Aspaveli	Swedish Orphan Biovitrum AB (publ),SE-112 76 Stockholm,Sweden	Authorised	12/13/2021

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
SYFOVRE pegcetacoplan 15 mg/0.1 mL solution for injection vial	404733	Apellis Australia Pty Ltd	Medicine	A	1/29/2025
EMPAVELI pegcetacoplan 1080 mg/20 mL injection solution vial	346216	Swedish Orphan Biovitrum Pty Ltd	Medicine	A	2/3/2022

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
EMPAVELI	Swedish Orphan Biovitrum AB (Publ)	02533294	Solution - Subcutaneous	1080 MG / 20 ML	2/15/2023

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
ASPAVELI 1080 MG SOLUCION PARA PERFUSION	Swedish Orphan Biovitrum Ab (Publ)	1211595001	SOLUCIÓN PARA PERFUSIÓN	Diagnóstico Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

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