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Evidence: HighPeptide conjugateFDA ApprovedPegylated cyclic peptideFDA-approved 2021 · Empaveli2023 · Syfovre (eye)

Pegcetacoplan

A complement C3 blocker for a blood disease, two kidney diseases and an eye disease

Pegcetacoplan joins two cyclic peptides to a PEG chain and blocks complement protein C3. As Empaveli it treats PNH and two kidney diseases; as Syfovre, injected into the eye, it slows geographic atrophy.

At a Glance Read Research →
Overview

What is it?

Complement is a cascade of blood proteins that tags and destroys microbes, and several diseases come from it attacking the body's own tissue. Older complement drugs such as eculizumab block C5 near the end of the cascade; pegcetacoplan blocks C3 at its centre, which also stops the clearance of coated red cells outside blood vessels.

In paroxysmal nocturnal haemoglobinuria (PNH), red cells lack protection from complement and are destroyed. In PEGASUS, patients still anaemic on eculizumab who switched to pegcetacoplan gained 3.84 g/dL more haemoglobin by week 16. Approved as Empaveli in 2021, it was later approved for C3 glomerulopathy and immune-complex MPGN after the VALIANT trial cut urine protein by 68% relative to placebo.

As Syfovre it is injected into the eye. In OAKS and DERBY it slowed the growth of geographic atrophy, the advanced dry form of macular degeneration, by up to 22% at two years, without improving vision tests. Empaveli carries a boxed warning for serious infections from encapsulated bacteria; Syfovre's label warns of endophthalmitis, retinal vasculitis and intraocular inflammation.

Structured data

At a Glance

Every value in this table is printed from the Grey Peptides dataset, the same record the compound explorer and comparison tool read, so the three can never disagree.

ClassTwo identical cyclic peptides joined by a 40 kDa polyethylene glycol chain, which bind complement protein C3; FDA-approved as Empaveli (2021, under the skin) for PNH and later C3G/IC-MPGN, and as Syfovre (2023, into the eye) for geographic atrophy.
FormulaC1970H3848N50O947S4
Molecular weight43500 g/mol
Half-life8.6 days (subcutaneous, PNH); 10.2 days (C3G/IC-MPGN); 4.5 days (intravitreal, geographic atrophy)
Label dose1080 mg (twice weekly (Empaveli); 15 mg per eye every 25 to 60 days (Syfovre), subcutaneous infusion; intravitreal)
US statusFDA Approved
Evidence levelHigh
Last reviewed2026-10-03

Educational information, not medical advice. The doses on this page come from approved labels, published studies and, where no study exists, amounts reported by users. None is a recommendation for you. An unapproved compound has no established safe or effective human dose, and products sold for “research use only” are not made or tested for people. Talk to a doctor before acting on anything on this site, including before you start, stop or change any medicine or dose. Full medical disclaimer.

Interactive

Half-Life: How Much Remains

The dataset records a half-life of 8.6 days (subcutaneous, PNH); 10.2 days (C3G/IC-MPGN); 4.5 days (intravitreal, geographic atrophy) for Pegcetacoplan. After each half-life, half of what was in the circulation is gone, so about 3% remains after five. Real clearance varies with the person, the dose, the route and kidney or liver function; this is arithmetic on a published figure, not a dosing tool.

Time since a single dose
ElapsedTimeRemaining
1 half-life8.6 days50% remaining
2 half-lives17.2 days25% remaining
3 half-lives25.8 days12.5% remaining
4 half-lives34.4 days6.25% remaining
5 half-lives43 days3.125% remaining
Evidence

Published Research

The studies behind pegcetacoplan, read from their PubMed abstracts, with dosing from both labels read October 3, 2026.

Human2021

PEGASUS: against eculizumab in PNH

In 80 adults still anaemic on eculizumab, pegcetacoplan raised haemoglobin by 3.84 g/dL more by week 16, and 85% against 15% no longer needed transfusions. Injection-site reactions (37% against 3%) were more common, breakthrough haemolysis less common.

PMID: 33730455
Human2025

Three years in PNH

Across PEGASUS, PRINCE and their extension, haemoglobin and fatigue stayed near normal for up to three years, with yearly transfusion avoidance of 71% to 86%; breakthrough haemolysis occurred in 28%.

PMID: 40720060
Human2024

PNH with milder anaemia

In 25 patients whose haemoglobin was already 10 g/dL or more, 16 weeks raised it to about 12.7 to 14.0 g/dL.

PMID: 39079163
Human2025

VALIANT: C3G and IC-MPGN

In 124 adolescents and adults, urine protein fell 67.2% on pegcetacoplan against a 2.9% rise on placebo at 26 weeks; 49% against 3% met a composite kidney end point. A biopsy activity score did not differ significantly.

PMID: 41337715
Human2023

OAKS and DERBY: geographic atrophy

In 1,258 patients, injections slowed lesion growth at 24 months by 16% to 22% against sham; DERBY missed its 12-month end point. Vision did not differ, and new wet macular degeneration occurred in 6% to 13% of treated patients against 2% to 4% on sham.

PMID: 37865470
Human2025

GALE: 36 months in the eye

In an open-label extension retaining 92% of 790 patients, lesion growth was reduced by up to 32% against projected sham, more each year.

PMID: 40280279
Safety

Side Effects & Contraindications

Reported Side Effects

From the studies above.

● Injection-site reactions and diarrhoea (Empaveli)
● Breakthrough haemolysis in PNH
● New wet macular degeneration (Syfovre)

Contraindications & Cautions

Warnings from the Empaveli (DailyMed, effective August 21, 2026) and Syfovre (effective September 16, 2025) labels.

● Boxed warning: serious infections caused by encapsulated bacteria; vaccination required, and Empaveli is available only through a REMS
● Monitoring for PNH manifestations after stopping Empaveli
● Interference with some laboratory tests
● Syfovre: endophthalmitis, retinal detachment, retinal vasculitis or vascular occlusion, wet macular degeneration, intraocular inflammation and raised eye pressure
Questions

Frequently Asked Questions

?What is pegcetacoplan?

Two cyclic peptides joined by a polyethylene glycol chain that block complement protein C3, sold as Empaveli for PNH and two kidney diseases and as Syfovre for geographic atrophy.

?How is it different from eculizumab?

Eculizumab blocks C5, late in the complement cascade; pegcetacoplan blocks C3, earlier, which also stops red cell destruction outside vessels.

?Does Syfovre improve vision?

No. In OAKS and DERBY it slowed lesion growth but did not improve vision tests over two years.

Bibliography

References

  1. [fda] US FDA, Drugs@FDA (openFDA): EMPAVELI (pegcetacoplan), NDA 215014, original approval May 14, 2021; SYFOVRE (pegcetacoplan), NDA 217171, original approval February 17, 2023; both prescription. Read October 3, 2026.
  2. [fda-pi] FDA. Empaveli (pegcetacoplan) US prescribing information (DailyMed set c23d89e9-b00b-4520-e053-2995a90a95af, effective August 21, 2026). Read October 3, 2026.
  3. [fda-pi] FDA. Syfovre (pegcetacoplan) US prescribing information (DailyMed set b4434e61-9ec9-4e0e-bd60-45aaa0a87e64, effective September 16, 2025). Read October 3, 2026.
  4. [clinical-trial] Hillmen P, et al. "Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria." N Engl J Med, 2021;384(11):1028-1037. PMID: 33730455.
  5. [clinical-trial] de Castro C, et al. "Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan." Adv Ther, 2025;42(9):4641-4658. PMID: 40720060.
  6. [clinical-trial] Panse J, et al. "Improvements in hematologic markers and decreases in fatigue with pegcetacoplan for patients with paroxysmal nocturnal hemoglobinuria and mild or moderate anemia (hemoglobin ≥10 g/dL) who had received eculizumab or were naive to complement inhibitors." PLoS One, 2024;19(7):e0306407. PMID: 39079163.
  7. [clinical-trial] Fakhouri F, et al. "Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN." N Engl J Med, 2025;393(22):2210-2220. PMID: 41337715.
  8. [clinical-trial] Heier JS, et al. "Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials." Lancet, 2023;402(10411):1434-1448. PMID: 37865470.
  9. [clinical-trial] Wykoff CC, et al. "Pegcetacoplan Treatment for Geographic Atrophy in Age-Related Macular Degeneration Over 36 Months: Data From OAKS, DERBY, and GALE." Am J Ophthalmol, 2025;276:350-364. PMID: 40280279.

Sources & Citations

Studies were read from their PubMed records; regulatory sources are listed below, read October 3, 2026.

Medical Disclaimer: This content is for educational and informational purposes only. It is not medical advice, and nothing here is an instruction to obtain or use any compound. Consult a licensed healthcare provider before making health decisions.

Last reviewed: 2026-10-03