Pegcetacoplan Injection
Pegcetacoplan Injection is a complement inhibitor used to treat paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN).
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 2 clinical label sets within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
⚠️ FDA Boxed Warning — the FDA’s strongest safety alert ▾
Empaveli can cause serious, life-threatening infections from certain bacteria. Because Empaveli suppresses part of the immune system (the complement system), it greatly increases the risk of dangerous infections — especially from bacteria such as Neisseria meningitidis (meningitis), Streptococcus pneumoniae (pneumonia), and Haemophilus influenzae type B. These infections have been fatal in some patients, even in those who were vaccinated. You must be up to date on vaccines against these bacteria at least 2 weeks before your first dose, unless your doctor determines the urgency of treatment outweighs that wait. Even after vaccination, stay alert for warning signs: sudden fever, severe headache, stiff neck, sensitivity to light, chills, or body aches — and get emergency care immediately if these occur. Because of this risk, Empaveli is only available through a special restricted program called the Empaveli REMS.
📖 Pegcetacoplan Injection: Patient Information Guide
A plain-language walkthrough of Pegcetacoplan Injection — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Pegcetacoplan injection is used across two different products to treat distinct rare conditions involving immune system overactivation.
- Empaveli (subcutaneous): Treats adults with paroxysmal nocturnal hemoglobinuria (PNH) — a rare disorder where the complement immune system destroys the patient's own red blood cells
- Empaveli (subcutaneous): Treats adults and children age 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) — rare kidney diseases — to reduce protein leaking into the urine and slow kidney function loss
- Syfovre (intravitreal): Administered by a qualified physician directly into the eye — product-specific; not interchangeable with Empaveli's uses or route
⚙️How it works▾
Pegcetacoplan targets a key protein in the complement system — a part of the immune system that normally helps fight infection but can go haywire in certain diseases. It binds to complement protein C3 and its activated form, C3b, essentially putting the brakes on a chain reaction that would otherwise destroy red blood cells (in PNH) or deposit damaging fragments in kidney filters (in C3G and IC-MPGN). By acting early in this cascade, it controls both types of red blood cell destruction seen in PNH and reduces the complement deposits that drive kidney disease in C3G and IC-MPGN.
💊How it's taken▾
Empaveli is administered as a slow infusion under the skin (subcutaneous), typically twice weekly. Syfovre is injected directly into the eye by a qualified physician, once every 25 to 60 days per affected eye — the physician prepares the syringe immediately before use, and each vial treats only one eye.
- Follow your prescriber's schedule exactly — do not skip or delay doses without guidance
- If you miss a dose, contact your care team for instructions rather than doubling up
- No specific food restrictions are mentioned, but discuss your full medication list with your prescriber
🤒Side effects — in detail▾
The most commonly reported side effects differ somewhat between conditions but include:
- Injection or infusion site reactions — redness, swelling, bruising, itching, or pain at the site
- Infections, including respiratory tract and viral infections
- Diarrhea and abdominal pain (more common in PNH patients)
- Fatigue
- Fever (pyrexia)
- Headache and dizziness
- Nausea
- Cough
More serious reactions — including life-threatening bacterial infections, severe allergic reactions (anaphylaxis), and, after stopping, a rebound of red blood cell destruction in PNH — require immediate medical attention. Do not wait to call your doctor if you notice sudden fever, severe headache, stiff neck, or difficulty breathing.
⚠️Warnings & precautions▾
- Life-threatening bacterial infections: Empaveli suppresses the complement immune system, dramatically raising the risk of serious infections from encapsulated bacteria like meningococcus, pneumococcus, and Haemophilus influenzae type B. These can be fatal within hours. Vaccination is required before starting, but does not fully protect you — stay alert for fever, stiff neck, severe headache, or sensitivity to light and seek emergency care immediately.
- Empaveli REMS program: Because of this infection risk, Empaveli is only dispensed through a restricted safety program. Your prescriber must be enrolled, and you'll receive a Patient Safety Card to carry at all times — including for 2 months after stopping.
- Rebound hemolysis after stopping Empaveli in PNH: Stopping treatment can trigger a rapid return of red blood cell destruction. Your doctor will monitor you for at least 8 weeks after your last dose.
- Infusion-related reactions: Allergic reactions including anaphylaxis have occurred — if you experience facial swelling, hives, or trouble breathing during an infusion, stop and get emergency help.
- Lab test interference: Empaveli can interfere with silica-based blood clotting tests (aPTT), causing falsely abnormal results. Always tell any lab you are on this medication.
- Syfovre eye injection risks: Eye infections (endophthalmitis) and elevated eye pressure can occur after intravitreal injection — report eye pain, redness, light sensitivity, or vision changes promptly.
🔀What it interacts with▾
The FDA label identifies a limited but important interaction to be aware of:
- Silica-based coagulation test reagents: Empaveli can interfere with certain blood clotting lab tests that use silica reagents, making results appear falsely prolonged (aPTT). Inform any clinician or laboratory performing clotting studies that you are taking Empaveli so they can use an alternative testing method.
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Vaccination is required: You must receive up-to-date vaccines against meningococcus, pneumococcus, and Haemophilus influenzae type B at least 2 weeks before starting Empaveli — unless your doctor determines the urgency of treatment outweighs the wait
- Active serious infection: Empaveli cannot be started if you currently have an unresolved serious encapsulated bacterial infection
- Pregnancy: Animal studies showed increased risk of miscarriage and stillbirth at higher doses; discuss the benefits and risks with your doctor before starting. Use effective contraception during treatment and for 40 days after the last dose.
- Breastfeeding: Breastfeeding should be stopped during treatment and for 40 days after the last dose due to potential risk to the infant
- Children: Empaveli is approved for C3G/IC-MPGN in patients 12 and older, but safety and effectiveness in children under 12 or in pediatric PNH patients have not been established
- Older adults: Clinical studies did not include enough patients 65 and older to fully characterize differences — your prescriber will monitor you appropriately
- Eye conditions (Syfovre): Syfovre must not be used if you have an active eye or surrounding-tissue infection, active inflammation inside the eye, or a known allergy to pegcetacoplan or any Syfovre ingredient
- Immunosuppressive medications: Concurrent use with immunosuppressants may further increase infection risk — make sure your prescriber knows all medications you take
📡Pharmacodynamics — effects in the body▾
In PNH patients, pegcetacoplan substantially reduced the proportion of red blood cells coated with the damaging C3b protein (dropping from roughly 18% to near zero), while increasing the number of healthy PNH red blood cells in circulation. In C3G and primary IC-MPGN patients, it markedly raised low complement C3 levels in the blood and reduced levels of a harmful downstream immune complex (C5b-9) that contributes to kidney injury; 74% of patients on pegcetacoplan showed a dramatic reduction in complement deposits in kidney tissue by Week 26, compared to only 12% on placebo. At recommended doses, pegcetacoplan did not cause any significant changes to heart rhythm (QTc interval).
🔬Pharmacokinetics — how your body processes it▾
After a subcutaneous dose, pegcetacoplan reaches peak blood levels in roughly 4.5 to 6 days. Steady-state concentrations are achieved in about 4 to 8 weeks with regular dosing. The drug has a relatively long effective half-life — about 8.6 to 10.8 days depending on the condition being treated — meaning it stays active in the body for an extended period after each dose. Pegcetacoplan is broken down into small peptides and amino acids through normal metabolic pathways, and its pharmacokinetics are not meaningfully affected by age, sex, race, kidney impairment, or liver function within the ranges studied.
📦How to store it▾
Refrigerate SYFOVRE between 2°C to 8°C (36°F to 46°F). Store the vial in the original carton to protect from light.
📄 Written from Pegcetacoplan Injection’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
🩺 Pharmacist Counseling Corner
Quick, plain-English answers to the questions patients ask most about Pegcetacoplan Injection — the kind of thing our pharmacy team would talk through with you at the counter.
Why do I have to get vaccinations before starting Empaveli? Can't I just start right away? ▾
What does the injection site reaction feel like, and is it something to worry about? ▾
What happens if I decide to stop taking Empaveli? ▾
I'm pregnant or planning to become pregnant. Is Empaveli safe? ▾
I have some blood tests coming up. Does Empaveli affect lab results? ▾
What's the Patient Safety Card I heard about, and do I really need to carry it? ▾
Is Pegcetacoplan Injection available over the counter? ▾
When was Pegcetacoplan Injection first available? ▾
💬 Answers drafted from Pegcetacoplan Injection’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Pegcetacoplan Injection comes
Pegcetacoplan Injection is available in 1 form. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Injection
How this form is used
- Empaveli is used via subcutaneous (under-the-skin) infusion to treat adults with paroxysmal nocturnal hemoglobinuria (PNH)
- Empaveli is used via subcutaneous infusion to treat adults and children 12 and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria and slow kidney function loss
- Syfovre is administered by intravitreal injection (into the eye) by a qualified physician
- Empaveli is given as a subcutaneous infusion, typically twice weekly — follow your prescriber's exact schedule
- Syfovre is injected into each affected eye by a qualified physician once every 25 to 60 days; only 0.1 mL is given per eye per dose
- Syfovre vials should be stored in the refrigerator and allowed to reach room temperature for at least 15 minutes before use; each vial is for single-eye use only
- After any Syfovre injection, report eye pain, redness, light sensitivity, or blurred vision to your doctor without delay
📊 Pegcetacoplan Injection across the U.S. market
How Pegcetacoplan Injection appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
📈 How widely Pegcetacoplan Injection is used
A general measure of how commonly Pegcetacoplan Injection is prescribed, based on Medicaid — one of the largest public drug programs. The figure below is prescriptions filled in Q1–Q4 2025, summed across every form and brand of the drug.
Think of this as a proxy for how widely Pegcetacoplan Injection is used, drawn from freely available public data. Medicaid is just one program — these numbers don’t include Medicare, other government coverage, or commercial and cash-pay prescriptions, so real-world use is higher than what’s shown here.
How commonly is Pegcetacoplan Injection prescribed? Of the 1,596 medications we measure, Pegcetacoplan Injection ranks #988 by Medicaid prescriptions — more than 38% of them.
Utilization by Pegcetacoplan Injection product
Pick a product to see its own Medicaid numbers. Each chip is one clinical product — a specific strength & dosage form as defined by RxNorm — with brand-name and generic versions combined.
0.1 ML pegcetacoplan 150 MG/ML Injection Most dispensed
Summed across the 1 of 4 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 2630847
20 ML pegcetacoplan 54 MG/ML Injection
Summed across the 1 of 1 listed NDC products with Medicaid activity — every brand and generic of this exact strength & form. RxNorm 2557386
Shares are of the Medicaid prescriptions shown here. Product grouping follows RxNorm (the National Library of Medicine’s drug terminology), so “one product” means one strength & dosage form regardless of manufacturer. Dollar figures are gross Medicaid reimbursement before mandatory rebates — rebates (often large, especially for brand-name drugs) are confidential, so actual net cost to Medicaid is lower than shown.
Source: Medicaid State Drug Utilization Data (CMS), aggregated by generic ingredient — summed across every NDC (all brands, strengths, salt forms and dosage forms) of Pegcetacoplan Injection, and across all states and both fee-for-service and managed-care claims. A general popularity signal; it excludes Medicare, commercial insurance and cash prescriptions, so it is not total U.S. use. Based on the 2 of 5 listed Pegcetacoplan Injection NDCs with Medicaid activity.
🔍 Compare Pegcetacoplan Injection products
A representative set of FDA-listed product records for Pegcetacoplan Injection — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Injection | ||||
| 15 mg/0.1 mL | Intravitreal | Apellis Pharmaceuticals, Inc. × 2 listings | NDA | View NDC → |
| 1080 mg/20 mL | Subcutaneous | Apellis Pharmaceuticals, Inc. × 3 listings | NDA | View NDC → |
Showing 5 of 5 products — FDA National Drug Code Directory. See every product, package size and price: browse all 5 Pegcetacoplan Injection NDCs →
📈 What people report — real-world data (FDA FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Pegcetacoplan Injection — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Pegcetacoplan Injection in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
🏛️ The road to your pharmacy
How Pegcetacoplan Injection went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 Recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
Source: openFDA drug enforcement (recall) reports.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2557372 1 dose form · 2 strengths
-
Dose form (SCDF) Injection RxCUI 2557385In current FDA listingsClinical drug / strength (SCD) 0.1 ML pegcetacoplan 150 MG/MLRxCUI 263084720 ML pegcetacoplan 54 MG/MLRxCUI 2557386
Look up RxCUI 2557372 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
📦 Supply & shortage status
Whether Pegcetacoplan Injection is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
RxNorm drug class
Pegcetacoplan Injection belongs to the Complement Inhibitor class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
🔬 Where this comes from
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 2 FDA-filed SPL label sets within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. Apellis Pharmaceuticals, Inc. is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.