Pegcetacoplan dosage and evidence
Written by Aaron CuhaReviewed Sep 2026
The short answer
Pegcetacoplan is one drug with two unrelated approvals.
Evidence grade for Pegcetacoplan: Strong. The rule: at least two finished studies in people, and at least 1,000 people who took it. The sum on this page: 3 completed trials, 1,258 people given it. A grade counts how much has been done to people, not whether it works.
- People given it
- 1,258
- Human studies
- 3
- Found nothing
- 0
Dose
Approved medicine. No human dose established for unlabelled uses
Labeled amounts sit on the prescribing information. This page records what trials measured, not a personal dose.
FDA-approved twice. Empaveli (NDA 215014) was approved 14 May 2021 as a drip under the skin. It is for adults with paroxysmal nocturnal haemoglobinuria, a rare disease where the immune system destroys red blood cells. It is now also approved from age 12 for two rare kidney diseases, C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis. Syfovre (NDA 217171) was approved 17 February 2023 as a shot into the eye. It is for geographic atrophy, a late stage of age-related macular degeneration. The European Medicines Agency refused the geographic atrophy use in 2024.
3 effects reported, 3 unwanted, by people using it, not a trial
Goal pages: Gut health
In plain English
In plain English, from Aaron
This is how I would explain it to a friend. The evidence, with the numbers, is further down the page.
Pegcetacoplan is one drug with two jobs. As Empaveli, a drip under the skin, it treats PNH. That is a rare disease where the immune system bursts red blood cells. As Syfovre, a shot into the eye, it treats a late stage of age-related sight loss. It works by blocking part of the immune system.
What people take it for
- Treating PNH when the older drug is not enough.
- Slowing the late, dry form of macular degeneration.
- Two rare kidney diseases.
What studies in people showed
In the eye, 1258 people took part in two studies. The damaged patch grew 16 to 22 percent more slowly by two years. But sight was no better. The wet form of the disease started more often on the drug. Europe refused the eye use for that reason. In 80 people with PNH who were still anaemic on the older drug, it raised haemoglobin by 3.84 g/dL more. 85 percent avoided a blood transfusion, against 15 percent.
What people report
Reports, not study results
The good
- We have not collected user reports on this one yet.
The bad
- In the PNH study, 37 percent had reactions where the drip went in, and 22 percent had diarrhoea.
- The label warns about serious infections, and vaccines are needed first.
- In the eye studies, the wet form of macular degeneration started more often on the drug.
Where these come from: We have no user reports. The side effects are from the studies and the label on this page.
My bottom line
For PNH, it is a real win you can feel. For the eye, it slows a scan, not your sight loss. That is a talk with your eye doctor.
An opinion, not a finding. I am a coach, not a doctor.
What it has been measured to do
Measured in people
Goals Pegcetacoplan has been tested for in people, biggest studies first. We count only what the studies on this page say, so the real numbers may be higher.
- Eyes and vision1,258 people · 1 study
- Gut and digestionno headcount stated · 1 study
34 of the 36 indexed goals have no study of any kind behind Pegcetacoplan
No study in people, animals or cells on this page reports a result for these. That does not prove Pegcetacoplan does nothing for them. It does mean anything you have heard about Pegcetacoplan and one of these did not come from a study cited here.
Show full evidence
What is Pegcetacoplan?
Pegcetacoplan is one drug with two unrelated approvals. As Empaveli, a drip under the skin, it treats a rare blood disease called PNH. There it beat the older drug eculizumab, raising haemoglobin by 3.84 g/dL more at 16 weeks. As Syfovre, a shot into the eye, it treats a late form of age-related vision loss. There it slowed the damaged patch from growing by 16 to 22 percent, but sight was no better at 24 months. European regulators refused the eye use for exactly that reason.
Pegcetacoplan is two identical small ring-shaped proteins, joined by a long chain of a plastic-like material called PEG. Most of its weight is the PEG. It blocks C3, a key part of the complement system, which is part of your immune defence. C3 sits one step above C5, the target of the older drugs eculizumab and ravulizumab.
In the blood, where it blocks matters. PNH is a rare disease where the immune system attacks red blood cells. Blocking C5 stops red cells bursting inside blood vessels. But it leaves bits of C3 stuck to the red cells, and the liver and spleen then remove them. So some people on eculizumab stay anaemic. Blocking C3 stops both kinds of damage. The FDA approved Empaveli on 14 May 2021 for adults with PNH. It has since been approved for two rare kidney diseases, C3 glomerulopathy and a related condition, from age 12. There it is used to cut protein in the urine and slow loss of kidney function.
The eye is a different story with a different verdict. The FDA approved Syfovre on 17 February 2023 for geographic atrophy, a late stage of age-related macular degeneration. It is given as a shot into the eye. Approval rested on two 24-month studies, OAKS and DERBY. They measured how fast the damaged patch at the back of the eye grew, on scans. The drug slowed that growth. At 12 months, DERBY missed its main goal, with only 12 percent slowing for monthly shots. Both studies showed slowing by 24 months. The published paper says, in one sentence, that there were no differences in the main vision tests at 24 months.
That sentence is why European regulators said no in June 2024, and confirmed it on review in September 2024. Their reasoning was that slowing the patch did not give patients a benefit they could feel. They said a treatment's benefits should show up in everyday life. Two regulators looked at the same two studies and drew opposite conclusions about whether a slower-growing patch counts as a benefit. That disagreement is the most useful thing on this page.
How does Pegcetacoplan work?
It sticks to C3, and to C3b, a piece of it, and stops C3 being split. That blocks the step shared by all three routes the complement system uses. Because C3 sits above C5, blocking it does two things. It stops the attack that bursts red cells in the blood. It also stops C3b tagging red cells for removal by the liver and spleen. In the eye, one route of the complement system is linked to the slow, local inflammation that comes with geographic atrophy. A shot into the eye puts the drug right there. How broad the block is also the risk. Taking out C3 removes more immune defence than taking out C5. The label warns about serious infections from certain bacteria with a protective coat.
What have studies in people found about Pegcetacoplan?
Two finished sets of final-stage studies, in two unrelated diseases. In PNH, the gain in haemoglobin and the drop in blood transfusions are large and easy to see. In geographic atrophy, the effect is on a patch measured on a scan. No benefit to sight was shown over 24 months.
- OAKS and DERBY, 1258 people: growth of the damaged patch slowed 16 to 22 percent at 24 months. DERBY missed its 12-month goal.
- No differences in the main vision tests at 24 months. That covers sharpness of sight, reading speed, and being able to read on your own.
- The wet form of macular degeneration started more often on pegcetacoplan than on the fake injection, in both eye studies.
- PEGASUS, 80 people: haemoglobin was 3.84 g/dL higher at week 16 on pegcetacoplan than on eculizumab.
- 85 percent avoided a blood transfusion on pegcetacoplan, against 15 percent on eculizumab.
- PEGASUS did not show it was as good on LDH, the standard blood marker of red cells bursting inside blood vessels. That is the expected cost of blocking C3 instead of C5.
What this does not tell you: PEGASUS only took people already anaemic on eculizumab. So it says nothing about people never treated before. It had 80 patients and everyone knew which drug they got. That matters for things patients rate themselves, like tiredness. The eye studies measured a scan result. A slower-growing patch is a stand-in for sight, and sight itself did not differ. Blocking C3 takes out more of the immune system than blocking C5, and the long-term infection risk is not fully known. The maker paid for both sets of studies.
The state of the research
This page shows what a fight over stand-in measures looks like when two capable regulators disagree in public.
The FDA approved Syfovre because the damaged patch grew more slowly. The European regulators refused it because, in their words, benefits should affect patients' everyday lives, and that was not shown. Both were reading OAKS and DERBY. They do not disagree about the data. They disagree about whether a change on an eye scan counts as a benefit when reading speed, reading on your own and sharpness of sight did not move over two years. The European review was decided with some members voting against, so the committee itself was split.
Two more facts belong in any honest account. DERBY, one of the two studies, missed its main goal at 12 months. And the wet form of macular degeneration, a separate and serious problem, started more often on pegcetacoplan than on the fake injection in both studies. That is a real trade-off, not background noise.
The blood use is a different matter and should not be judged the same way. A haemoglobin gain of 3.84 g/dL, and transfusion-free rates rising from 15 to 85 percent, are results patients feel directly. Even there, the study reported one goal it did not meet. Reporting that is the standard this archive holds itself to.
The evidence, in charts
Fig. 1a · how many people
1,258people, across 1 study in people cited here
| Study | People | Share |
|---|---|---|
| The Lancet 2023 | 1,258 | 100% |
The whole total is one study of 1,258. Counts are the number of people each study on this page says it signed up. That includes studies that only watched people and gave nobody the compound. 3 more studies in people give no headcount and are not counted here. Studies still signing people up are left out, and only studies cited on this page are counted.
Fig. 1b · evidence mix
All 4 sources here are studies in people.
- Study in people · done in people4100%
Counted from the list of sources on this page. It counts only what we cite, so there may be more studies out there. A review counts once, however many studies it sums up.
Fig. 2 · evidence over time
The studies cover 3 different years, 2021 to 2025, counted from the list of sources on this page.
Fig. 3 · legal status at a glance
US
Approved
UK
Approved
AU
Approved
CA
Approved
Approved in all four countries shown. This is what the law says, not a verdict on the science. See Legal status below for the full text and any notes.
Fig. 5 · what the molecule is
- Kind of compound
- Peptide
- Molecule weight
- About 43.5 kDa, dominated by the 40 kDa PEG linker rather than by the two 13-residue peptides.
- Half-life
- About 8.6 daysVery long, weeks+
- Chain length
- None on file
Half-life as stated on file: About 8.6 days as the median effective half-life in paroxysmal nocturnal haemoglobinuria. Reported as longer in C3 glomerulopathy and shorter after intravitreal injection, where the geometric mean is roughly four and a half days.
No amino acid sequence is on file for Pegcetacoplan, so none is drawn rather than guessed from a name.
The studies we cite
- Study in people2021Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
Pegcetacoplan beat the existing drug in people whose blood disease it was failing to control. 80 adults who were still anaemic on eculizumab were switched to one drug or the other, and everyone knew which. Haemoglobin rose clearly more on pegcetacoplan. 35 of 41 on it avoided a transfusion, against 6 of 39 on eculizumab. One blood marker of cell destruction did not come out as well. Injection site reactions and diarrhoea were much more common on the new drug. Nobody in either group got meningitis.
PEGASUS. Phase 3, open-label, randomised, in adults with paroxysmal nocturnal haemoglobinuria and haemoglobin below 10.5 g/dL despite eculizumab. 41 to pegcetacoplan monotherapy, 39 to eculizumab. Primary endpoint, change in haemoglobin at week 16, favoured pegcetacoplan by an adjusted least-squares mean difference of 3.84 g/dL, P below 0.001. 35 of 41 (85 percent) on pegcetacoplan avoided transfusion versus 6 of 39 (15 percent). Non-inferiority was shown for change in reticulocyte count but NOT for change in lactate dehydrogenase. Injection site reactions 37 versus 3 percent and diarrhoea 22 versus 3 percent; breakthrough haemolysis 10 versus 23 percent. No meningitis in either group.
- Study in people2023Pegcetacoplan 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
The eye injection slowed the damaged area from spreading and did nothing for anyone's sight. 1,258 people aged 60 and over took part in two identical trials, one of which hit its one year target and one of which missed. By two years both showed the damage growing roughly a fifth more slowly on treatment. Vision itself was no better. Treatment also triggered the wet form of the disease far more often, in roughly one in ten against one in fifty. Funded by Apellis.
1258 patients aged 60 and over. At 12 months OAKS met its primary endpoint, with lesion growth slowed 21 percent monthly (P equal to 0.0004) and 16 percent every other month (P equal to 0.0055), while DERBY missed it, at 12 percent (P equal to 0.062) and 11 percent (P equal to 0.085). By 24 months both were significant, 22 and 18 percent in OAKS and 19 and 16 percent in DERBY. The paper states there were no differences in key secondary visual function endpoints at 24 months. New-onset exudative age-related macular degeneration at 24 months: 11 and 8 percent on pegcetacoplan versus 2 percent on sham in OAKS, and 13 and 6 percent versus 4 percent in DERBY. Funded by Apellis Pharmaceuticals.
- Study in people2025Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan
The benefit lasted three years in the people who stayed on the drug. 114 of 133 patients from two earlier trials carried on into an extension study. Haemoglobin, fatigue scores and the markers of blood cell destruction improved within a month and stayed close to normal. In most years, between seven and nine in ten patients avoided a transfusion. Serious side effects hit more than half the group, though few were blamed on the drug. Five people died and none of those deaths were attributed to it.
Longer-term data out to three years in adults with paroxysmal nocturnal haemoglobinuria. This is an open-label extension, not a randomised comparison, so it describes what happens to patients who stayed on the drug rather than what the drug does relative to an alternative.
- Study in people2021Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The registry entry gives the size of the trial that changed how this disease is treated. It is marked completed and it enrolled 80 people. That is a very small trial to have moved practice. It also accepted only patients who were still anaemic while on the older drug, so it says nothing about anybody else.
The registry record for PEGASUS. Status COMPLETED, actual enrolment 80. Confirms the small size of the trial that changed practice in this disease, and that enrolment was restricted to patients already anaemic on eculizumab.
Where to buy Pegcetacoplan
Pegcetacoplan is an approved medicine. The approved product comes from a pharmacy, with a prescription. The vials below are sold “for research” and are not the approved drug.
No seller we track lists Pegcetacoplan yet. We read sellers’ public product lists every week, and this section fills in as soon as one does. Every seller, graded
Common questions
Has Pegcetacoplan been tested in people?
Yes. 1,258 people given it across 3 completed trials cited here, 0 of which found nothing. Pegcetacoplan is one drug with two unrelated approvals.
What is the Pegcetacoplan dosage?
Approved medicine. No human dose established for unlabelled uses. Labeled amounts sit on the prescribing information. This page records what trials measured, not a personal dose. FDA-approved twice. Empaveli (NDA 215014) was approved 14 May 2021 as a drip under the skin. It is for adults with paroxysmal nocturnal haemoglobinuria, a rare disease where the immune system destroys red blood cells. It is now also approved from age 12 for two rare kidney diseases, C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis. Syfovre (NDA 217171) was approved 17 February 2023 as a shot into the eye. It is for geographic atrophy, a late stage of age-related macular degeneration. The European Medicines Agency refused the geographic atrophy use in 2024.
Is Pegcetacoplan FDA-approved?
FDA-approved twice. Empaveli (NDA 215014) was approved 14 May 2021 as a drip under the skin. It is for adults with paroxysmal nocturnal haemoglobinuria, a rare disease where the immune system destroys red blood cells. It is now also approved from age 12 for two rare kidney diseases, C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis. Syfovre (NDA 217171) was approved 17 February 2023 as a shot into the eye. It is for geographic atrophy, a late stage of age-related macular degeneration. The European Medicines Agency refused the geographic atrophy use in 2024.
Why does the same drug have two brand names?
Because it holds two unrelated approvals, given different ways at different doses. Empaveli is a drip under the skin for PNH, a rare blood disease, and now also C3 glomerulopathy, a rare kidney disease. Syfovre is a shot into the eye for geographic atrophy. Same molecule, different product, different doctor, different specialty.
Does Syfovre improve vision?
No. The studies measured how fast the damaged patch grew on eye scans. That slowed by 16 to 22 percent at 24 months. On the vision tests, including sharpness of sight, reading speed and reading on your own, there were no differences at 24 months. That gap is the whole argument about this drug.
Why did Europe reject it when the FDA approved it?
European regulators said no in June 2024, and confirmed it on review in September 2024. Their reasoning was that slowing the patch did not give patients a benefit that matters, and that benefits should show up in everyday life. The FDA accepted the scan result. Same studies, different judgment about what counts as a benefit.
Is pegcetacoplan better than eculizumab?
In the people PEGASUS studied, adults still anaemic on eculizumab, it gave 3.84 g/dL more haemoglobin at week 16. It lifted the share avoiding transfusion from 15 to 85 percent. It did not show it was as good on LDH, a blood marker of red cells bursting. It has not been tested against eculizumab in people never treated with this kind of drug.
What are the main risks?
Blocking C3 takes out more immune defence than blocking C5. So the label warns about serious infections from certain coated bacteria, and requires vaccines first. In PEGASUS, 37 percent had reactions where the drip went in, and 22 percent had diarrhoea. For the eye product, the warnings include infection inside the eye, a detached retina, and inflamed or blocked blood vessels in the eye. The wet form of macular degeneration also started more often than with the fake injection.