Empaveli

(Pegcetacoplan)

ApprovedPeptide

Pegcetacoplan is used to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH), where the immune system mistakenly attacks and destroys red blood cells, causing anemia and other serious complications.[2] Empaveli is currently approved and in use across 14 indications[4].[1]

Empaveli is one of 103 peptide programs targeting Dowling-Degos disease 1. It is currently in Approved.

Indications

Total Trials

Active Trials

102

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Dowling-Degos Disease 1Active, not recruiting

C3 GlomerulonephritisActive, not recruiting

Complement 3 GlomerulopathyActive, not recruiting

Eye DiseaseActive, not recruiting

Membranoproliferative GlomerulonephritisCompleted

Dense Deposit DiseaseActive, not recruiting

Atrophic Macular DegenerationApproved

Focal Segmental GlomerulosclerosisApproved

Primary Peritoneal CancerRecruiting

C3 Glomerulopathy (C3G)Recruiting

Dowling-Degos Disease 1Approved

Active, not recruiting

C3 GlomerulonephritisApproved

Active, not recruiting

Complement 3 GlomerulopathyApproved

Active, not recruiting

Eye DiseaseApproved

Active, not recruiting

Membranoproliferative GlomerulonephritisPhase 3

Completed

Dense Deposit DiseaseApproved

Active, not recruiting

Atrophic Macular DegenerationApproved

Approved

Focal Segmental GlomerulosclerosisApproved

Approved

Primary Peritoneal CancerPhase 2

Recruiting

C3 Glomerulopathy (C3G)Approved

Recruiting

Showing 10 of 14 indications

Competition

Competitive Landscape

130 competitors in Safety and Efficacy

#DrugCompanyPhaseTrials

Empaveli

Apellis Pharmaceuticals

Approved

2 2

Avastin

Roche

Phase 3

3 3

Actemra

Roche

Phase 3

1 4

Humira

RRanbaxy Laboratories Limited

Phase 3

3 5

Rayos

RRoxane Laboratories

Phase 3

3 6

Fludara

Sanofi

Phase 2

5 7

CAB LA

GSK

Phase 3

1 8

Yervoy

Bristol-Myers Squibb

Phase 2

3 9

B/F/TAF

Gilead

Phase 3

5 10

Eloxatin

Teva

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Active, not recruiting

Age-Related Macular Degeneration

NCT06161584

Q3 2027

Phase 3Active, not recruiting

Membranoproliferative Glomerulonephritis

NCT05809531

Q2 2028

Phase 2Recruiting

Primary Peritoneal Cancer

NCT04919629

Q4 2028

PreclinicalRecruiting

Metastatic Pancreatic Ductal Adenocarcinoma

NCT07214298

Phase 2Recruiting

Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT04901936

Clinical

Trials by Indication[1]

Membranoproliferative Glomerulonephritis2 trials

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

Active, not recruiting· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 3·NCT05809531·100 enrolled

Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 3·NCT05067127·124 enrolled

Age-Related Macular Degeneration2 trials

A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy

Active, not recruiting· Boehringer Ingelheim logo

Boehringer Ingelheim·Phase 2·NCT06722157·202 enrolled

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

Active, not recruiting· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals··NCT06161584·300 enrolled

Safety and Efficacy2 trials

Pegcetacoplan Long Term Safety and Efficacy Extension Study

Active, not recruiting· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 3·NCT03531255·160 enrolled

Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN

Active, not recruiting· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 2·NCT04572854·13 enrolled

Geographic Atrophy2 trials

Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 2·NCT02503332·246 enrolled

Study to Evaluate the Safety of Intravitreal APL-2 in Patients Diagnosed With Geographic Atrophy

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 1·NCT03777332·19 enrolled

Paroxysmal Nocturnal Hemoglobinuria2 trials

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 3·NCT03500549·80 enrolled

A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Active, not recruiting·SSwedish Orphan Biovitrum··NCT05776472·165 enrolled

16 more indications

CAR1 trial

A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 1·NCT02264639·9 enrolled

Efficacy1 trial

A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 2·NCT03593200·4 enrolled

Wet AMD1 trial

Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 1·NCT02461771·13 enrolled

Cold Agglutinin Disease1 trial

A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)

Completed·SSwedish Orphan Biovitrum·Phase 3·NCT05096403·24 enrolled

Neovascular AMD1 trial

Pegcetacoplan (APL-2) in Neovascular AMD

Terminated· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 1/2·NCT03465709·17 enrolled

IC-MPGN1 trial

C3G/Primary IC-MPGN EAP

Approved for marketing· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals··NCT04729062

Primary Peritoneal Cancer1 trial

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

Recruiting·RRoswell Park Cancer Institute·Phase 2·NCT04919629·60 enrolled

Thrombotic Microangiopathy1 trial

A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation

Completed·SSwedish Orphan Biovitrum·Phase 2·NCT05148299·12 enrolled

Amyotrophic Lateral Sclerosis1 trial

MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)

Terminated· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 2·NCT04579666·249 enrolled

Pilot Study1 trial

Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 1·NCT02588833·23 enrolled

C3 Glomerulopathy1 trial

A 2-year, Single-arm, Multi-center Study to Explore the Efficacy and Safety of Pegcetacoplan in Preventing Disease Recurrence in Adolescent and Adult Kidney Transplant Recipients With C3 Glomerulopathy or Primary Immune-complex Membranoproliferative Glomerulonephritis

Not yet recruiting·SSwedish Orphan Biovitrum·Phase 3·NCT07898774·30 enrolled

Geographic Atrophy Secondary to Age-related Macular Degeneration1 trial

A Phase 3, Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Completed· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 3·NCT04770545·792 enrolled

C3 Glomerulopathy (C3G)1 trial

Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy (C3G) or Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN): A Multi-Country Study

Recruiting·SSwedish Orphan Biovitrum·Approved·NCT07746895·150 enrolled

Delayed Graft Function1 trial

A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation

Withdrawn· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 3·NCT07020832

Metastatic Pancreatic Ductal Adenocarcinoma1 trial

A Phase I/II Study of Complement Inhibition in Pancreatic Ductal Adenocarcinoma

Recruiting·RRoswell Park Cancer Institute·Preclinical·NCT07214298·35 enrolled

Paroxysmal Nocturnal Hemoglobinuria (PNH)1 trial

An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

Recruiting· Apellis Pharmaceuticals logo

Apellis Pharmaceuticals·Phase 2·NCT04901936·12 enrolled

Regulatory

FDA Approval Timeline[5]

May 14, 2021

EMPAVELI

NDA NDA215014

May 14, 2021

EMPAVELI

NDA NDA215014

Feb 8, 2023

EMPAVELI

NDA NDA215014

Feb 17, 2023

SYFOVRE

NDA NDA217171

Sep 28, 2023

EMPAVELI

NDA NDA215014

Feb 8, 2024

EMPAVELI

NDA NDA215014

Oct 7, 2024

EMPAVELI

NDA NDA215014

Jun 10, 2025

EMPAVELI

NDA NDA215014

Jul 28, 2025

EMPAVELI

NDA NDA215014

Aug 20, 2026

EMPAVELI

NDA NDA215014

Sources

References

[1]
ClinicalTrials.gov — 26 trial records for Pegcetacoplan. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Empaveli drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Pegcetacoplan compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Pegcetacoplan. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA215014). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Empaveli. U.S. Food & Drug Administration.
[7]
PubMed — 1 publication indexed for Pegcetacoplan. U.S. National Library of Medicine.
[8]
SEC EDGAR — Apellis Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: C3 Complement Inhibitor[3]
Modality: Peptide[3]
Highest Phase: Approved[1]
Company: Apellis Pharmaceuticals

Patent Landscape[6]

Patent Cliff

Apr 7, 2027

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9056076Oct 25, 2026

8168584Apr 7, 2027

9169307Nov 18, 2027

7888323Dec 4, 2027

7989589Dec 4, 2027

11 more patents

11661441Jan 13, 2033

10125171Aug 2, 2033

10035822Nov 15, 2033

11292815Nov 15, 2033

10875893Nov 15, 2033

12528836Oct 7, 2036

11903994Feb 22, 2037

11040107Apr 9, 2038

11844841Dec 9, 2038

12458695Dec 14, 2038

12290566Dec 14, 2038

Key Publications[7]

Pegcetacoplan for Geographic Atrophy: OAKS and DERBY Phase 3 Results

LancetSep 22, 2023DOI: 10.1016/S0140-6736(23)01735-7

Empaveli

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q3 2027

Q2 2028

Q4 2028

Trials by Indication[1]

Membranoproliferative Glomerulonephritis2 trials

Age-Related Macular Degeneration2 trials

Safety and Efficacy2 trials

Geographic Atrophy2 trials

Paroxysmal Nocturnal Hemoglobinuria2 trials

CAR1 trial

Efficacy1 trial

Wet AMD1 trial

Cold Agglutinin Disease1 trial

Neovascular AMD1 trial

IC-MPGN1 trial

Primary Peritoneal Cancer1 trial

Thrombotic Microangiopathy1 trial

Amyotrophic Lateral Sclerosis1 trial

Pilot Study1 trial

C3 Glomerulopathy1 trial

Geographic Atrophy Secondary to Age-related Macular Degeneration1 trial

C3 Glomerulopathy (C3G)1 trial

Delayed Graft Function1 trial

Metastatic Pancreatic Ductal Adenocarcinoma1 trial

Paroxysmal Nocturnal Hemoglobinuria (PNH)1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

Key Publications[7]

Related News