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Pegcetacoplan (Empaveli, Syfovre)_Dosage, benefits & legal status

Also known as Empaveli, Aspaveli, Syfovre, APL-2

Published October 14, 2026

Blocks complement at its central protein, C3. Twice-weekly infusions for PNH and kidney disease; eye injections every one to two months for geographic atrophy.

Block complement attackComplement C3 inhibitor (PEGylated cyclic peptide)FDA-approvedHigh-risk
InImmune support
Dose1,080 mg twice weekly under the skin; 15 mg into the eye every 25 to 60 days
FrequencyTwice weekly (Empaveli); every 25 to 60 days (Syfovre)
RouteUnder the skin by pump or on-body injector (Empaveli); into the eye (Syfovre)
StatusFDA-approved
MixYour choice, open the Pegcetacoplan (Empaveli, Syfovre) calculator
CycledContinuous, open-ended

Common vial size is what vendors typically sell, not a recommendation. Enter your own vial in the calculator.

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01

What is Pegcetacoplan (Empaveli, Syfovre)?

Plain language first. The technical label stays, but it never stands alone.

Overview

Under the skin, as Empaveli, it treats paroxysmal nocturnal haemoglobinuria (PNH), where complement destroys red blood cells; against eculizumab it raised haemoglobin by 2.37 g/dL while the eculizumab group's fell by 1.47. Since 2025 it also treats two complement-driven kidney diseases, C3 glomerulopathy and primary IC-MPGN. Injected into the eye as Syfovre, it slowed the growth of geographic atrophy by about a fifth but did not improve vision tests, and the EU and UK refused it.

The technical version

Pegcetacoplan is two identical 14-amino-acid cyclic peptides, compstatin analogues closed by a disulfide between cysteines 2 and 12, each joined through its last lysine to one end of a 40 kDa PEG chain; the whole molecule weighs about 43.5 kDa. It binds complement C3 and its active fragment C3b, blocking all three complement pathways where they meet. Compstatin came from John Lambris's laboratory at the University of Pennsylvania; Apellis developed pegcetacoplan and licensed the systemic drug outside the US to Sobi.

What it's used for

Prescription only: twice-weekly infusions under the skin for PNH, C3 glomerulopathy or primary IC-MPGN (from age 12 for the kidney diseases), or an eye injection for geographic atrophy, every 25 to 60 days in the US and every other month in Australia.

02

How does Pegcetacoplan (Empaveli, Syfovre) work?

What it does in the body, and where.

Pegcetacoplan blocks complement at C3, the step all three activation routes pass through, so it acts earlier than C5 blockers.

  1. 01
    The hub
    Enzyme complexes called convertases cut complement protein C3 into C3a and C3b. C3b coats targets for removal and builds more convertase, including the one that cuts C5 and starts the membrane attack complex.
  2. 02
    Blocking C3
    Pegcetacoplan binds C3 and C3b, so C3 is not cut and C3b cannot build new convertases. In PNH this stops red cells being punched open in the blood and also stops C3b-coated red cells being removed by the liver and spleen; C5 blockers stop only the first.
  3. 03
    The PEG chain
    The 40 kDa PEG chain slows clearance, giving an effective half-life of 8.6 to 10.8 days, long enough for twice-weekly dosing. In the eye it damps local complement activity thought to drive the loss of retinal cells.
Pathway
THE PEPTIDE
Pegcetacoplan (Empaveli, Syfovre)
ACTS ON
Complement C3 and C3b, the hub of all three complement pathways
RESULT
In PNH, red cells are neither punched open nor tagged for removal
RESULT
Less C3 builds up in the kidney filters, and complement activity in the retina is damped

A schematic of the compound's mechanism, not a diagnostic image.

03

What else is it used for?

Each group carries its own evidence level.

Approved means regulators have cleared it for that purpose. Investigational means it is in trials. Early research means it is being studied and is not established.

PNH

FDA-approved
  • Human trialPEGASUS, 80 adults switching from eculizumab: haemoglobin difference 3.84 g/dL at 16 weeks; transfusion avoidance 85% against 15%.
  • Human trialPRINCE, 53 adults never treated with a complement inhibitor: haemoglobin stabilised in 85.7% against 0% on supportive care.
  • No dataPEGASUS was open-label, and pegcetacoplan did not show non-inferiority to eculizumab on LDH.
Study
Hillmen P et al., NEJM 2021

Kidney disease

FDA-approved
  • Human trialVALIANT: proteinuria 68.1% lower than placebo at 26 weeks; 60% against 5% halved their protein loss.
  • No dataThe paper's testing order stopped at kidney biopsy scores, where there was no difference, so eGFR was not formally tested; the US label added loss of kidney function to the indication in August 2026.
Study
Fakhouri F et al., NEJM 2025

Geographic atrophy

FDA-approved
  • Human trialAt 24 months lesion growth was 22% and 18% slower in OAKS and 19% and 16% slower in DERBY (monthly and every other month).
  • No dataDERBY missed its 12-month primary endpoint, and neither trial improved visual function; the EU and UK refused approval on that basis.
Study
Heier JS et al., Lancet 2023

ALS

tested-and-failed
  • Human trialMERIDIAN, 249 people for 52 weeks: missed its primary and key secondary endpoints; development stopped in May 2023.
Often left out

'Raised haemoglobin by 3.84 g/dL' is the gap between groups: pegcetacoplan rose 2.37 and eculizumab fell 1.47. The 22% and 18% slowing at 24 months is OAKS only; DERBY was 19% and 16%, and DERBY missed its 12-month primary endpoint. 'Up to 36%' is a selected 6-month window in one arm. Syfovre has not been shown to slow vision loss; that is why the EU and UK refused it. The label calls each arm a pentadecapeptide because it counts a non-amino-acid spacer as position 14; each arm has 14 amino acids. Empaveli, Aspaveli and Syfovre are the same molecule at very different doses: 1,080 mg under the skin against 15 mg in the eye. The half-life is 8.6 days in PNH but 10.2 to 10.8 days in the kidney diseases. In VALIANT, kidney function was not formally tested in the paper, though the US label now names it.

Study
Apellis, MERIDIAN top-line results (2023)

The published record

Research index

373

Papers and trials about Pegcetacoplan (Empaveli, Syfovre)

227 papers tagged human · 120 not reviews or lab · 39 clinical trial publications

36 registered trials · 15 with posted results

Furthest stage reached: Approved in the US

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In animals12
In the lab1
Reviews120
Senior author shareSpread across many groups

Evidence summary

Counted from Europe PMC indexing, not read by a person; a human tag can land on reviews, commentary or lab papers; clinical trial publications is the narrower count.

Pegcetacoplan (Empaveli, Syfovre) vial rendering
Compound field notes

At a glance

Four things to know about Pegcetacoplan (Empaveli, Syfovre)

01

Evidence

FDA-approved

02

Category

Immune support

03

Common vial

Varies

04

Half-life

8.6 days in PNH; 10.2 to 10.8 days in kidney disease

04

How much, and do you cycle it?

The range, and whether you take breaks.

1,080 mg twice weekly under the skin; 15 mg into the eye every 25 to 60 days, twice weekly (empaveli); every 25 to 60 days (syfovre). Continuous, open-ended FDA label

Both products are long-term treatments. In PNH, stopping risks red cell breakdown, so monitoring continues for at least 8 weeks afterwards.

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05

How long does it stay in the body?

The half-life, and where the figure comes from.

8.6 days in PNH; 10.2 to 10.8 days in kidney diseaseFDA label

Effective half-life after injection under the skin. Peak levels 4.5 to 6 days after a single dose, with steady state in 4 to 8 weeks. Broken down into small peptides and amino acids. After an eye injection only small amounts reach the blood, with a systemic half-life of about 4.5 days.

Source: Empaveli US prescribing information section 12.3; Syfovre US prescribing information section 12.3

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06

What does it cost?

Typical dose and cost, in plain numbers.

Pegcetacoplan (Empaveli, Syfovre) is not reconstituted

It is not supplied as a powder measured in milligrams, so there is no water-to-powder math and no draw to calculate here. Doses are written in mg, from a ready-made solution.

Typical dose: 1,080 mg twice weekly under the skin; 15 mg into the eye every 25 to 60 days

Empaveli: 1,080 mg (one 20 mL vial) under the skin twice weekly by infusion pump (about 30 minutes over two sites) or the single-use on-body Empaveli Injector; in PNH, every 3 days if LDH rises above twice the upper limit. Teenagers with kidney disease under 50 kg get weight-banded doses of 540 to 810 mg. When switching from eculizumab, both run together for 4 weeks. Syfovre: 15 mg (0.1 mL) into each affected eye every 25 to 60 days, by an eye specialist. Vaccination against encapsulated bacteria at least 2 weeks before starting Empaveli. Both come as ready-made solutions, so there is nothing to mix.

07

How do you store it?

Before mixing, and after.

Dry powder in the freezer or fridge. Once mixed, fridge, and use within about a month.

Unmixed lyophilized Pegcetacoplan (Empaveli, Syfovre) keeps for a long time cold. Once you add water the clock starts: most reconstituted vials hold up for roughly 28 to 30 days at fridge temperature. Keep it out of light, and do not freeze it after mixing.

08

What are the side effects and cautions?

Known cautions and warnings for this compound.

Solubility, computed: 36% charged residues, 50% hydrophobic. Water usually sufficient.

A first-pass rule from peptide manufacturers' guidance. It does not account for disulfide bridges, salt form or how the powder was dried, and it predicts what should be easy rather than what will happen in your vial.

Who should avoid it

Check these before anything else.
  • Unresolved serious infection by encapsulated bacteria (meningococcus, pneumococcus, Haemophilus influenzae type b): Empaveli must not be started
  • No vaccination against these bacteria at least 2 weeks before the first infusion, unless delay is riskier and antibiotics are given meanwhile
  • Syfovre: infection in or around the eye, or active inflammation inside the eye
  • Allergy to pegcetacoplan or its ingredients; anaphylaxis has been reported
  • Pregnancy and breastfeeding: high doses raised miscarriage and stillbirth in monkeys, and breastfeeding should stop during treatment and for 40 days after

Stop and get help

These are emergencies. Seek care.
  • Fever, chills, headache with a stiff neck or a rash: possible serious infection, an emergency even after vaccination
  • After an eye injection: sudden loss of vision, eye pain, worsening redness or light sensitivity, possible retinal vasculitis, infection or detachment
  • Swelling of the face or throat, hives or trouble breathing
  • Do not stop Empaveli for PNH on your own: red cell breakdown can return, and the label asks for at least 8 weeks of monitoring after stopping

Common effects

Expected, and usually mild.
  • PNH (PEGASUS, 16 weeks): injection-site reactions in 39% against 5% on eculizumab, diarrhoea in 22% against 3%, abdominal pain in 20% against 10%
  • Infections in 29% against 26% in PNH; one death from septic shock in the PRINCE trial
  • Kidney disease (VALIANT, 26 weeks): fever in 19% against 10% on placebo, nasopharyngitis in 18% against 12%, influenza in 11% against 5%
  • Eye (OAKS and DERBY, 2 years): new wet macular degeneration in 12% (monthly) and 7% (every other month) against 3% with sham injections
  • Eye: floaters in 10% and 7% against 1%; intraocular inflammation in 4% and 2% against under 1%
  • Antibodies against the peptide in 29% of kidney-trial patients over 52 weeks

Where this page says nothing is established, that means nobody has studied it, not that a compound is safe.

09

Is Pegcetacoplan (Empaveli, Syfovre) approved?

And where the numbers on this page come from.

Yes. Pegcetacoplan (Empaveli, Syfovre) is FDA approved and available on prescription in the United States. It is also approved in the European Union, the United Kingdom, Japan, and Australia.

What has happened recently

Two products share one ingredient. Empaveli (Aspaveli in the EU), under the skin: FDA-approved for adults with PNH on 14 May 2021 (NDA 215014, priority review, orphan drug), with a boxed warning for encapsulated-bacteria infections and a REMS; extended on 28 July 2025 to C3 glomerulopathy and primary IC-MPGN from age 12, with the August 2026 label adding the loss of kidney function to the aim. The EU authorised Aspaveli on 13 December 2021 (Sobi holds it), at first only for patients still anaemic on a C5 inhibitor, and added the kidney diseases on 16 January 2026; Great Britain authorised it on 24 February 2022 and now covers the kidney diseases too. Japan approved it for PNH in March 2023 and the kidney diseases in August 2026. Syfovre, into the eye: FDA-approved on 17 February 2023 (NDA 217171) for geographic atrophy, with a retinal vasculitis warning added in November 2023, and approved in Australia in January 2025. The EU refused Syfovre in 2024 after re-examination, finding no meaningful benefit on everyday vision against the injection risks, and the UK's MHRA rejected it in January 2025. Sobi paid $250 million upfront in 2020 for ex-US rights to the systemic drug. No generic exists.

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10

What else is like Pegcetacoplan (Empaveli, Syfovre)?

Others in Immune support, side by side.

If you're weighing Pegcetacoplan (Empaveli, Syfovre), it's worth reading Pexiganan, Splenopentin, and Thymopentin (TP-5) in the same class.

YOU ARE HERE
Pegcetacoplan (Empaveli, Syfovre)

Blocks complement at its central protein, C3. Twice-weekly infusions for PNH and kidney disease; eye injections every one to two months for geographic atrophy.

FDA-approved
Dose
1,080 mg twice weekly under the skin; 15 mg into the eye every 25 to 60 days
How often
Twice weekly (Empaveli); every 25 to 60 days (Syfovre)
PEXI
Pexiganan

A magainin-based antimicrobial cream, tested twice for diabetic foot infections and never approved.

Investigational
Dose
0.8% cream twice daily (last trials)
How often
Twice daily for 14 days (trials)
SPLENO
Splenopentin

A five-amino-acid spleen peptide, sold on an evidence base almost nobody has read.

Research only
Dose
Research only
How often
Not established
TP-5
Thymopentin (TP-5)

Five amino acids that carry the whole activity of a thymus hormone.

Clinical use
Dose
50 mg per dose
How often
3x per week
See other immune support

Guides for this compound

Sources

Beyond these, 373 records are indexed. Browse the records