Back to discover
Drug

Pegcetacoplan

Approved medicine
ClassComplement C3 inhibitorResearchActive indication expansion

Also known as Empaveli, Syfovre.

RxNorm2557372ChEMBLCHEMBL4298211UNIITO3JYR3BOU
7
Active clinical trials
Complement C3
Primary target
9
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

Two-Year Experience with Intravitreal Pegcetacoplan for the Treatment of Geographic Atrophy in Clinical Practice

Industry2026-09-10American Journal of Ophthalmology

Early Versus Delayed Pegcetacoplan Treatment for Geographic Atrophy Secondary to Age-Related Macular Degeneration in OAKS, DERBY, and GALE

Industry2026-08-28American Journal of Ophthalmology

Indication expansion: PEGCETACOPLAN (NDA215014)

Regulatory2026-08-20FDA

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

Clinical trial2026-08-19Results expected Q1 2028 · ClinicalTrials.gov

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

Clinical trial2026-06-26Results expected Q4 2028 · ClinicalTrials.gov

Real-world outcomes of pegcetacoplan treatment in C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis

Industry2026-06-16Kidney International

Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of ABBV-6628 in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Clinical trial2026-06-15Results expected Q4 2029 · ClinicalTrials.gov

An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Clinical trial2026-05-19Completed · ClinicalTrials.gov

Randomized, Double-masked, Active-controlled, Multicenter Study to Evaluate Efficacy and Safety of Two Regimens of Intravitreal BI 771716 Against Pegcetacoplan in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration VERDANT Trial

Clinical trial2026-05-14Results expected Q4 2026 · ClinicalTrials.gov

Indication expansion: PEGCETACOPLAN (NDA215014)

Regulatory2025-07-28FDA

Label change: PEGCETACOPLAN (NDA215014)

Regulatory2025-06-10FDA

Supplemental approval: PEGCETACOPLAN (NDA215014)

Regulatory2024-10-07FDA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Pegcetacoplan
Aliases & brands
EmpaveliSyfovre
RxNorm CUI
2557372
ChEMBL ID
CHEMBL4298211
UNII
TO3JYR3BOU
Primary mechanism
Complement C3 inhibitor
Regulatory jurisdictions
emafda

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Complement C3
C3 · P01024
INHIBITORComplement C3 inhibitor

Regulatory timeline

9 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Approval / market entry confirmed across FDA and EMA (2021-05-14 → 2023-02-08)
Earliest approval
2021-05-14
Latest approval
2026-08-20
Authorities
FDA · EMA
Total events
9
fdaU.S. Food and Drug Administration· 8 events
2026-08-20Indication expansion
Indication expansion: PEGCETACOPLAN (NDA215014)
Evidence ↗
2025-07-28Indication expansion
Indication expansion: PEGCETACOPLAN (NDA215014)
Evidence ↗
2025-06-10Label change
Label change: PEGCETACOPLAN (NDA215014)
Evidence ↗
emaEuropean Medicines Agency· 1 event
2021-12-13ApprovalMulti-authority
Approval: Aspaveli (EMA)
Indication: Aspaveli is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. Aspaveli is indicated for the… Show full indication

Aspaveli is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. Aspaveli is indicated for the treatment of adult and adolescent patients aged 12 to 17 years with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) in combination with a renin-angiotensin system (RAS) inhibitor, unless RAS inhibitor treatment is not tolerated or contraindicated.

Evidence ↗

Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

23 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
23
registered trials across all phases
LATEST COMPLETION 2026
7
Active studies
5
Recruiting
7
Late-stage (III+)
13
Completed
3
Discontinued
PHASE DISTRIBUTIONn = 23
Phase 15Phase 1 / 23Phase 28Phase 37

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

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

SuspendedPH IIIApellis Pharmaceuticals, Inc.n=3202030

An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

Active, not recruitingPH IIIApellis Pharmaceuticals, Inc.n=1002027

An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

CompletedPH IIIApellis Pharmaceuticals, Inc.n=1372026

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

CompletedPH IIIApellis Pharmaceuticals, Inc.n=7922025

A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

CompletedPH IIIApellis Pharmaceuticals, Inc.n=1242025

Recent completions

Trials that read out recently, adding to the completed evidence base.

An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

CompletedPH IIIApellis Pharmaceuticals, Inc.n=1372026

An Open-Label, Randomized, Controlled, Phase 2 Study to Evaluate the Safety and Efficacy of Pegcetacoplan in the Treatment of Post-Transplant Recurrence of C3G or IC-MPGN

CompletedPH IIApellis Pharmaceuticals, Inc.n=132026

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

CompletedPH IIIApellis Pharmaceuticals, Inc.n=7922025

A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

CompletedPH IIIApellis Pharmaceuticals, Inc.n=1242025

An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients With Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

CompletedPH IISwedish Orphan Biovitrumn=122024

A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)

CompletedPH IIISwedish Orphan Biovitrumn=242024
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.