Empaveli (pegcetacoplan)
An approved treatment for C3 Glomerulopathy and Paroxysmal Nocturnal Hemoglobinuria.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Pegcetacoplan wherever you encounter it, plus the key facts at a glance.
- Generic name
- Pegcetacoplan
- Brand name
- Empaveli
- Development code
- APL-2
- Drug class
- C3 complement inhibitor
- Manufacturer
- Apellis Pharmaceuticals
- How it's taken
- Given as an infusion under the skin twice a week, using a commercially available infusion pump or the single-use EMPAVELI Injector worn on the belly.
A C3 complement inhibitor infused under the skin twice a week.
- The FDA first approved Empaveli on May 14, 2021 for adults with paroxysmal nocturnal hemoglobinuria (PNH), and on July 28, 2025 for adults and children 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN)[2].
- In an open-label trial in adults still anemic on eculizumab (Soliris), switching to Empaveli raised hemoglobin more than staying on eculizumab[1].
- In C3G, it reduced proteinuria by 68% compared with placebo in the VALIANT trial.
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Where Pegcetacoplan fits
One of two complement-targeted therapies now approved for C3G, alongside iptacopan (Fabhalta), a factor B inhibitor approved in March 2025.
- Pegcetacoplan blocks C3 (central amplification), while iptacopan blocks factor B (upstream in the alternative pathway).
- In PNH, Empaveli and iptacopan (Fabhalta) are the 2 complement inhibitors that act before C5 and can be used on their own, alongside the C5 inhibitors eculizumab (Soliris), ravulizumab (Ultomiris) and crovalimab (Piasky).
How Pegcetacoplan works
C3 glomerulopathy is caused by an overactive alternative complement pathway, a part of the immune system that normally helps fight infections. In C3G, this pathway spins out of control due to genetic mutations or autoantibodies (like C3 nephritic factor), depositing excessive amounts of complement protein C3 in the kidney's filtering units (glomeruli). This triggers chronic inflammation and progressive kidney damage.
Pegcetacoplan blocks C3, the central protein in the complement cascade where all three complement pathways converge and amplify. By stopping C3 activation, pegcetacoplan shuts down the entire amplification loop that drives C3 deposition in the kidneys. This is a broader block than targeting a single downstream component.
In the VALIANT trial (NCT05067127), pegcetacoplan reduced proteinuria (protein leakage into urine, a marker of kidney damage) by 68% compared with placebo. After 26 weeks, kidney function (eGFR) was 6.31 points higher on average with Empaveli than with placebo, and the label says it is used to reduce proteinuria and the loss of kidney function[1].
In PNH, red blood cells are missing protective proteins and are attacked in 2 ways. Complement can burst them inside blood vessels (intravascular hemolysis), and C3 fragments can coat them so the spleen and liver remove them (extravascular hemolysis). C5 inhibitors such as eculizumab and ravulizumab block only the first. The label says pegcetacoplan, acting at C3 earlier in the cascade, controls both[1].
Side effects and safety
- Hemolysis after stopping (PNH). Close monitoring for at least 8 weeks after stopping for signs such as high LDH, a drop in hemoglobin, dark urine, tiredness and blood clots.
- Infusion-related reactions. Allergic reactions such as facial swelling, rash, hives and fever, and cases of anaphylaxis that led to stopping treatment.
- Lab test interference. Can make the aPTT clotting test look falsely prolonged when silica reagents are used.
- Pneumococcal and meningococcal vaccination at least 2 weeks before the first dose
- Carry the Patient Safety Card during treatment and for 2 months after stopping
- In PNH, LDH checks twice a week for at least 4 weeks after any dose increase
- In PNH, watch for hemolysis for at least 8 weeks after stopping
Empaveli has a boxed warning: it raises the risk of serious, life-threatening infections from encapsulated bacteria (Streptococcus pneumoniae, Neisseria meningitidis and Haemophilus influenzae type B). Vaccines against these bacteria must be completed or updated at least 2 weeks before the first dose, and Empaveli is only available through a restricted safety program (the EMPAVELI REMS).
In the C3G and IC-MPGN trial, the most common side effects were infusion site reactions, fever, common cold symptoms (nasopharyngitis), flu, cough and nausea. In the PNH trial against eculizumab (Study APL2-302), side effects on Empaveli vs eculizumab included injection-site reactions (39% vs 5%), infections (29% vs 26%), diarrhea (22% vs 3%), belly pain (20% vs 10%), respiratory tract infection (15% vs 13%), viral infection (12% vs 8%) and tiredness (12% vs 23%), and serious side effects occurred in 17% of people on Empaveli[1].
People with PNH who stop Empaveli need monitoring for at least 8 weeks for returning hemolysis, and patients carry a Patient Safety Card during treatment and for 2 months after stopping[1]. Get medical care right away if you have signs of a serious infection. The label also warns about infusion-related reactions, including serious allergic reactions such as anaphylaxis, and Empaveli can make a clotting blood test (aPTT) look falsely prolonged when certain lab reagents are used.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Pegcetacoplan
Given as an infusion under the skin twice a week, using a commercially available infusion pump or the single-use EMPAVELI Injector worn on the belly. Patients or caregivers can give it at home after training. With a pump, an infusion takes about 30 minutes using 2 sites or about 60 minutes using 1 site; the Injector takes about 30 to 60 minutes. Vaccines against encapsulated bacteria (pneumococcal and meningococcal) are required at least 2 weeks before starting[1].
For PNH, adults get 1,080mg twice a week. If LDH, a blood marker of red cell destruction, rises above 2 times the upper limit of normal, the dose can change to 1,080mg every 3 days. People switching from eculizumab start Empaveli while staying on their eculizumab dose for 4 weeks, then stop eculizumab. People switching from ravulizumab start Empaveli no more than 4 weeks after their last ravulizumab dose[1].
For C3G or primary IC-MPGN, adults get 1,080mg twice a week, and children 12 to 17 get a dose based on body weight[1].
Prescribing Empaveli? REMS, monitoring and prior authorization criteria for prescribers
Availability and cost
Only available as the brand-name product.
Help paying for Empaveli
With private insurance, the drugmaker's copay card can lower what you pay for Empaveli. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE, but charity funds often can. Of the 6 charity funds for the conditions it treats, 5 were open on October 26, 2026. Without insurance, you can apply to the drugmaker's free medicine program, which sets its own eligibility rules. Pick your insurance below to see which offers fit you.
- Copay help
Eligible commercially insured patients may pay as little as $0 for EMPAVELI. Annual benefit limit; not for Medicare, Medicaid, CHIP, VA/DoD or TRICARE.
For: private insurance · source - Bridge or quick-start supply
The Apellis Bridge Program may give a temporary supply of EMPAVELI if your insurance changes or coverage is interrupted.
The official page does not say who qualifies. Ask the program. · source - Free medicine program
The Apellis Patient Assistance Program may help people with no insurance or limited coverage get EMPAVELI at no cost.
For: no insurance, underinsured · source - Insurance and case manager help
Care Coordinators explain insurance benefits and schedule deliveries; nurse educators train you to self-inject; vaccine support offered.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: Must enroll in ApellisAssist for the copay program. Terms and conditions apply to all programs.
Charity funds
- From a charity · NORD RareCarePNH Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for next year” - From a charity · NORD RareCarePNH Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for next year” - From a charity · NORD RareCareParoxysmal Nocturnal Hemoglobinuria (PNH) Emergency Relief fundOpen
Pays for: Emergency relief.
The foundation says: “Accepting Applications” - From a charity · The Assistance FundParoxysmal Nocturnal Hemoglobinuria (PNH) fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
- From a charity · TotalAssist (formerly PAN Foundation)Paroxysmal Nocturnal Hemoglobinuria fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).
- From a charity · Aplastic Anemia & MDS International FoundationPNH Patient Travel Assistance Fund fundApply directly
Pays for: Travel to a PNH specialist or second opinion (patient plus one companion), up to $800 per year.
The foundation says: “Status not shown on page”
Every program and charity fund for C3 Glomerulopathy →
Every program and charity fund for Paroxysmal Nocturnal Hemoglobinuria →
Clinical trial results
The Phase 3 VALIANT trial was the largest clinical trial ever conducted in C3 glomerulopathy and related conditions. Pegcetacoplan reduced proteinuria by 68% compared with placebo at week 26, meeting the primary endpoint. The FDA approved pegcetacoplan for C3G on July 28, 2025, making it one of the first approved treatments specifically for this rare kidney disease.
For PNH, Study APL2-302 (NCT03500549) enrolled 80 adults whose hemoglobin was still below 10.5g/dL after at least 3 months on eculizumab. After 4 weeks on both drugs, they were randomly assigned to Empaveli alone or eculizumab alone for 16 weeks. Hemoglobin rose 2.37g/dL on Empaveli and fell 1.47g/dL on eculizumab, a 3.84g/dL difference, and 85% vs 15% avoided transfusions. During that period, 3 people on Empaveli stopped because of hemolysis[1][3].
Study APL2-308 (NCT04085601) randomly assigned 53 adults who had not taken a complement inhibitor in the prior 3 months to Empaveli or supportive care such as transfusions for 26 weeks. Hemoglobin held steady without transfusion in 85.7% on Empaveli and 0% on supportive care, LDH fell by an average of 1,870U/L vs 400U/L, and 91% vs 6% avoided transfusions. Of the 18 people on supportive care, 11 switched to Empaveli after their hemoglobin dropped[1][4].
Development history
Apellis Pharmaceuticals developed pegcetacoplan as a C3-targeted complement inhibitor. The FDA first approved it as Empaveli for adults with paroxysmal nocturnal hemoglobinuria (PNH) on May 14, 2021[2]. The same molecule is sold as Syfovre, an eye injection approved in February 2023 for geographic atrophy, a form of macular degeneration.
The VALIANT trial expanded Empaveli's indications to C3 glomerulopathy, with FDA approval on July 28, 2025[2]. With iptacopan (a factor B inhibitor approved for C3G in March 2025), C3G now has 2 approved complement-targeted therapies.
Explore C3 Glomerulopathy and Paroxysmal Nocturnal Hemoglobinuria trials
Other C3 Glomerulopathy & Paroxysmal Nocturnal Hemoglobinuria treatments
Common questions about Pegcetacoplan
▸What is Pegcetacoplan (Empaveli)?
A C3 complement inhibitor infused under the skin twice a week. The FDA first approved Empaveli on May 14, 2021 for adults with paroxysmal nocturnal hemoglobinuria (PNH), and on July 28, 2025 for adults and children 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN)[2]. In an open-label trial in adults still anemic on eculizumab (Soliris), switching to Empaveli raised hemoglobin more than staying on eculizumab[1]. In C3G, it reduced proteinuria by 68% compared with placebo in the VALIANT trial.
▸How does Pegcetacoplan work?
C3 glomerulopathy is caused by an overactive alternative complement pathway, a part of the immune system that normally helps fight infections. In C3G, this pathway spins out of control due to genetic mutations or autoantibodies (like C3 nephritic factor), depositing excessive amounts of complement protein C3 in the kidney's filtering units (glomeruli). This triggers chronic inflammation and progressive kidney damage.
Pegcetacoplan blocks C3, the central protein in the complement cascade where all three complement pathways converge and amplify. By stopping C3 activation, pegcetacoplan shuts down the entire amplification loop that drives C3 deposition in the kidneys. This is a broader block than targeting a single downstream component.
In the VALIANT trial (NCT05067127), pegcetacoplan reduced proteinuria (protein leakage into urine, a marker of kidney damage) by 68% compared with placebo. After 26 weeks, kidney function (eGFR) was 6.31 points higher on average with Empaveli than with placebo, and the label says it is used to reduce proteinuria and the loss of kidney function[1].
In PNH, red blood cells are missing protective proteins and are attacked in 2 ways. Complement can burst them inside blood vessels (intravascular hemolysis), and C3 fragments can coat them so the spleen and liver remove them (extravascular hemolysis). C5 inhibitors such as eculizumab and ravulizumab block only the first. The label says pegcetacoplan, acting at C3 earlier in the cascade, controls both[1].
▸What are the side effects of Pegcetacoplan?
Empaveli has a boxed warning: it raises the risk of serious, life-threatening infections from encapsulated bacteria (Streptococcus pneumoniae, Neisseria meningitidis and Haemophilus influenzae type B). Vaccines against these bacteria must be completed or updated at least 2 weeks before the first dose, and Empaveli is only available through a restricted safety program (the EMPAVELI REMS). In the C3G and IC-MPGN trial, the most common side effects were infusion site reactions, fever, common cold symptoms (nasopharyngitis), flu, cough and nausea. In the PNH trial against eculizumab (Study APL2-302), side effects on Empaveli vs eculizumab included injection-site reactions (39% vs 5%), infections (29% vs 26%), diarrhea (22% vs 3%), belly pain (20% vs 10%), respiratory tract infection (15% vs 13%), viral infection (12% vs 8%) and tiredness (12% vs 23%), and serious side effects occurred in 17% of people on Empaveli[1]. People with PNH who stop Empaveli need monitoring for at least 8 weeks for returning hemolysis, and patients carry a Patient Safety Card during treatment and for 2 months after stopping[1]. Get medical care right away if you have signs of a serious infection. The label also warns about infusion-related reactions, including serious allergic reactions such as anaphylaxis, and Empaveli can make a clotting blood test (aPTT) look falsely prolonged when certain lab reagents are used.
▸How is Pegcetacoplan taken?
Given as an infusion under the skin twice a week, using a commercially available infusion pump or the single-use EMPAVELI Injector worn on the belly. Patients or caregivers can give it at home after training. With a pump, an infusion takes about 30 minutes using 2 sites or about 60 minutes using 1 site; the Injector takes about 30 to 60 minutes. Vaccines against encapsulated bacteria (pneumococcal and meningococcal) are required at least 2 weeks before starting[1].
For PNH, adults get 1,080mg twice a week. If LDH, a blood marker of red cell destruction, rises above 2 times the upper limit of normal, the dose can change to 1,080mg every 3 days. People switching from eculizumab start Empaveli while staying on their eculizumab dose for 4 weeks, then stop eculizumab. People switching from ravulizumab start Empaveli no more than 4 weeks after their last ravulizumab dose[1].
For C3G or primary IC-MPGN, adults get 1,080mg twice a week, and children 12 to 17 get a dose based on body weight[1].
▸Is Pegcetacoplan FDA approved?
Yes, Pegcetacoplan (Empaveli) is FDA approved (2021) for the treatment of C3 Glomerulopathy and Paroxysmal Nocturnal Hemoglobinuria.
▸What is the treatment for C3 glomerulopathy?
Two complement-targeted therapies are now FDA-approved for C3G: pegcetacoplan (Empaveli), a C3 inhibitor approved on July 28, 2025 for adults and children 12 and older, and iptacopan (Fabhalta), a factor B inhibitor approved in March 2025. [6]
▸How does Empaveli work for C3G?
Empaveli (pegcetacoplan) blocks complement C3, the central amplification protein in the complement cascade. In C3G, the complement system is overactive and deposits C3 in the kidneys. By blocking C3, Empaveli stops this damage at its source, and in the Phase 3 VALIANT trial it reduced proteinuria by 68% compared with placebo at week 26. [7][8]
▸How is Empaveli given?
Empaveli (pegcetacoplan) is given as an infusion under the skin twice a week using a small infusion pump or the single-use EMPAVELI Injector worn on the belly. Adults get 1,080mg per dose, children 12 to 17 get a weight-based dose, and each infusion takes about 30 to 60 minutes. Patients or caregivers can give it at home after training, and pneumococcal and meningococcal vaccines are required at least 2 weeks before starting. [8]
▸What are the side effects of Empaveli?
Empaveli (pegcetacoplan) has a boxed warning for serious, life-threatening infections from encapsulated bacteria (Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B), so vaccines must be completed at least 2 weeks before the first dose and the drug is only available through the EMPAVELI REMS safety program. In the C3G and IC-MPGN trial, the most common side effects were infusion site reactions, fever, common cold symptoms, flu, cough, and nausea. The label also warns about infusion-related reactions including anaphylaxis, and the drug can make the aPTT clotting test look falsely prolonged with certain lab reagents. [8][6]
▸Is Empaveli approved for PNH?
Yes. The FDA approved Empaveli for adults with paroxysmal nocturnal hemoglobinuria (PNH) on May 14, 2021, its first approval.
Who it is for: Adults with PNH, whether or not they have taken a C5 inhibitor before. The label says safety and effectiveness in children with PNH have not been established.
How it differs: It blocks C3, earlier in the complement cascade than Soliris, Ultomiris and Piasky, which block C5.
Source: FDA label for Empaveli, DailyMed, effective August 2026; Drugs@FDA
▸How is Empaveli dosed for PNH?
Empaveli is 1,080mg infused under the skin twice a week for adults with PNH.
Dose increase: If LDH rises above 2 times the upper limit of normal, the dose can change to 1,080mg every 3 days, with LDH checked twice a week for at least 4 weeks.
Missed dose: Give it as soon as possible, then return to the usual schedule.
At home: Patients or caregivers can give it with a pump or the EMPAVELI Injector after training; an infusion takes about 30 to 60 minutes.
Source: FDA label for Empaveli, DailyMed, effective August 2026
▸Empaveli vs Soliris for PNH: what did the trial show?
In a head-to-head trial in people still anemic on Soliris, Empaveli raised hemoglobin more.
Who was studied: The trial enrolled 80 adults with hemoglobin below 10.5g/dL after at least 3 months on eculizumab (Soliris).
Result at week 16: Hemoglobin rose 2.37g/dL on Empaveli and fell 1.47g/dL on eculizumab, and 85% vs 15% avoided transfusions.
Caveat: The trial was open label, and 3 people on Empaveli stopped during the 16 weeks because of hemolysis.
Source: FDA label for Empaveli, DailyMed, effective August 2026
▸Can I switch from Soliris or Ultomiris to Empaveli?
Yes, the label gives a switching plan for each.
From Soliris (eculizumab): Start Empaveli and keep your eculizumab dose for 4 weeks, then stop eculizumab.
From Ultomiris (ravulizumab): Start Empaveli no more than 4 weeks after your last ravulizumab dose.
Why the overlap: The label says it lowers the risk of hemolysis from stopping a C5 inhibitor abruptly.
Source: FDA label for Empaveli, DailyMed, effective August 2026
▸What are the side effects of Empaveli in PNH?
The most common in the PNH trials were injection-site reactions, infections, diarrhea and belly pain.
Rates vs eculizumab: Injection-site reactions 39% vs 5%, infections 29% vs 26%, diarrhea 22% vs 3%, belly pain 20% vs 10%.
Serious: Serious side effects occurred in 17% of people on Empaveli in the trial against eculizumab, and 1 person died of septic shock in the trial against supportive care.
Boxed warning: Serious infections from encapsulated bacteria; vaccines are required at least 2 weeks before starting.
After stopping: Monitoring for at least 8 weeks for returning hemolysis.
Source: FDA label for Empaveli, DailyMed, effective August 2026
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2026-08-21. EMPAVELI (pegcetacoplan) injection, for subcutaneous use: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c23d89e9-b00b-4520-e053-2995a90a95af
- U.S. Food and Drug Administration. Drugs@FDA: EMPAVELI, NDA 215014, approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215014
- ClinicalTrials.gov. Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH). https://clinicaltrials.gov/study/NCT03500549
- ClinicalTrials.gov. A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH. https://clinicaltrials.gov/study/NCT04085601
- Apellis Pharmaceuticals. Savings and support with EMPAVELI (PNH). https://empaveli.com/pnh/savings-and-support/
- Apellis Pharmaceuticals · July 28, 2025. FDA Approves Empaveli (pegcetacoplan) for C3 Glomerulopathy. https://www.globenewswire.com/news-release/2025/07/28/3122935/0/en/FDA-Approves-Apellis-EMPAVELI-pegcetacoplan-as-the-First-C3G-and-Primary-IC-MPGN-Treatment.html
- Apellis Pharmaceuticals. Pegcetacoplan in C3 Glomerulopathy, VALIANT Phase 3. https://apellis.com/our-science/pipeline/
- Apellis Pharmaceuticals. Empaveli (pegcetacoplan) for C3G. https://www.empaveli.com