Empaveli (pegcetacoplan)
An approved treatment for C3 Glomerulopathy.
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 small infusion pump or the single-use EMPAVELI Injector worn on the belly.
A C3 complement inhibitor approved for adults and children 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), based on the VALIANT trial, the largest ever conducted in this disease. Pegcetacoplan reduced proteinuria by 68% compared with placebo, blocking the overactive complement cascade at its central amplification point to prevent kidney damage.
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). Trials are comparing these mechanistic approaches. The availability of two distinct complement inhibitors represents a transformation in C3G treatment.
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, pegcetacoplan reduced proteinuria (protein leakage into urine, a marker of kidney damage) by 68%. This level of proteinuria reduction is associated with meaningful preservation of kidney function over time.
Mechanism: PEGylated peptide inhibitor of complement component C3, blocking the central amplification step of the complement cascade that drives glomerular inflammation and kidney damage in C3 glomerulopathy
Side effects and safety
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. 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 small infusion pump or the single-use EMPAVELI Injector worn on the belly. Adults get 1,080 mg per dose; children 12 to 17 get a dose based on body weight. Each infusion takes about 30 to 60 minutes, and patients or caregivers can give it at home after training. Treatment is ongoing. Vaccines against encapsulated bacteria (pneumococcal and meningococcal) are required at least 2 weeks before starting.
Availability and cost
Only available as the brand-name product.
Help paying for Empaveli
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- 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.
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.
Development history
Apellis Pharmaceuticals developed pegcetacoplan as a C3-targeted complement inhibitor. It was first approved for paroxysmal nocturnal hemoglobinuria (PNH) in 2021 and for geographic atrophy (a form of macular degeneration) in 2023. The VALIANT trial expanded its indications to C3 glomerulopathy, receiving FDA approval in July 2025. This approval alongside iptacopan (factor B inhibitor, approved March 2025) has transformed C3G from an untreatable condition to one with two mechanistically distinct targeted therapies.
Explore C3 Glomerulopathy trials
Other C3 Glomerulopathy treatments
Common questions about Pegcetacoplan
▸What is Pegcetacoplan (Empaveli)?
A C3 complement inhibitor approved for adults and children 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), based on the VALIANT trial, the largest ever conducted in this disease. Pegcetacoplan reduced proteinuria by 68% compared with placebo, blocking the overactive complement cascade at its central amplification point to prevent kidney damage.
▸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, pegcetacoplan reduced proteinuria (protein leakage into urine, a marker of kidney damage) by 68%. This level of proteinuria reduction is associated with meaningful preservation of kidney function over time.
▸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. 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 small infusion pump or the single-use EMPAVELI Injector worn on the belly. Adults get 1,080 mg per dose; children 12 to 17 get a dose based on body weight. Each infusion takes about 30 to 60 minutes, and patients or caregivers can give it at home after training. Treatment is ongoing. Vaccines against encapsulated bacteria (pneumococcal and meningococcal) are required at least 2 weeks before starting.
▸Is Pegcetacoplan FDA approved?
Yes, Pegcetacoplan (Empaveli) is FDA approved (2025) for the treatment of C3 Glomerulopathy.
▸What is the treatment for C3 glomerulopathy?
Two complement-targeted therapies are now FDA-approved for C3G: pegcetacoplan (Empaveli), a C3 inhibitor approved in July 2025, and iptacopan (Fabhalta), a factor B inhibitor approved in March 2025.
▸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.
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.
- 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