Pronunciation: em-PAH-vee-li
Generic name: pegcetacoplan
Dosage form: injection for subcutaneous use via an infusion pump or Empaveli injector (1080 mg/20 mL)
Drug class: Selective immunosuppressants

Medically reviewed by Carmen Pope, BPharm. Last updated on July 31, 2025.

What is Empaveli?

Empaveli is used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH) or to reduce proteinuria in adults and children 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN). It is given by subcutaneous infusion using an infusion pump or the on-body Empaveli injector.

Empaveli for PNH works by binding to specific proteins in the complement system, a part of the immune system that enhances (or complements) the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism, promote inflammation, and attack the pathogen's cell membrane. Empaveli binds to complement protein C3 and prevents it from splitting into C3a and C3b. This helps stop intravascular hemolysis (IVH, the destruction of red blood cells in the circulation) and extravascular hemolysis (EVH, the breakdown of red blood cells in the spleen, bone marrow, or liver by macrophages). Empaveli is the only treatment that targets C3 and may prevent both forms of red blood cell destruction (IVH and EVH). It works higher in the complement system than other treatments used for PNH.

In C3G and primary IC-MPGN, excessive complement activity causes harmful C3 fragment deposits in kidney filters, leading to damage. Empaveli targets C3 and C3b proteins, interrupting the complement pathway to prevent toxic accumulation and reduce renal inflammation and injury.

Empaveli (pegcetacoplan) gained FDA approval on May 14, 2021, for PNH. Approval was extended on July 28, 2025, to include C3G and IC-MPGN. There is no Empaveli generic. 

• Syfovre is an intravitreal formulation of pegcetacoplan that is used to treat an eye condition called geographic atrophy in adults with age-related macular degeneration (AMD). 

Empaveli side effects

Allergic reactions can happen during the administration of Empaveli for PNH. Stop your infusion and tell your healthcare provider or get emergency medical care right away if you get any of these symptoms during your infusion:

• facial swelling
• rash
• fever
• urticaria (hives)
• chest pain
• trouble breathing or shortness of breath
• swelling of your face, tongue, or throat
• feel faint or pass out.

The most common side effects occurring in 10% or more people on Empaveli for PNH are:

• injection-site reactions, such as redness, pain, or swelling at the injection site
• infections
• diarrhea
• pain in the stomach (abdomen)
• pain in the arms and legs
• low potassium levels
• tiredness
• cough
• joint pain
• dizziness
• headache
• a rash.

The most common side effects occurring in 10% or more people on Empaveli for C3G or primary IC-MPGN are:

• infusion-site reactions
• fever
• nasopharyngitis (a cold)
• Influenza
• cough
• nausea.

Tell your healthcare provider about any side effect that bothers you or that do not go away. These are not all of the possible side effects. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or to Apellis Pharmaceuticals, Inc. at 1-833-866-3346.

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Warnings

Empaveli can lower the ability of your immune system to fight infections and carries a Boxed Warning for increasing your risk of developing serious and life-threatening infections, including meningitis caused by encapsulated bacteria such as Streptococcus pneumoniae; Neisseria meningitidis, types A, C, W, Y, and B; and Haemophilus influenzae type B. You must be vaccinated against these bacteria at least 2 weeks before your first dose if you have not already had these vaccines. If your healthcare provider decides that urgent treatment with Empaveli is needed, you should receive the required vaccinations as soon as possible. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.

May cause infusion-related reactions or anaphylaxis, which may be life-threatening. Stop your infusion immediately and seek immediate medical attention if you develop any of the following: facial swelling, difficulty breathing, including shortness of breath and wheezing, swollen tongue or throat, feeling faint, rapid heart rate, skin reactions (such as a rash, hives, or itching), nausea or vomiting, confusion and anxiety, dizziness or fainting. 

After stopping Empaveli, patients with PNH will be closely monitored by their healthcare provider for at least 8 weeks for dangerous red blood cell breakdown (hemolysis). Warning signs include elevated LDH levels, fatigue, dark urine, stomach pain, breathing problems, blood clots, swallowing difficulties, or erectile dysfunction. If hemolysis occurs, consider restarting Empaveli treatment.

May interfere with laboratory tests, such as those that use silica reagents in coagulation panels (may cause an artificially prolonged activated partial thromboplastin time [aPTT]).

Vaccines reduce the risk of serious infections, but do not prevent all serious infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious infection once you have started taking Empaveli:

• fever with or without shivers or the chills
• fever and a rash
• shortness of breath
• extreme pain or discomfort
• headache with nausea or vomiting
• high heart rate
• headache and a fever
• headache with a stiff neck or stiff back
• confusion
• muscle aches with flu-like symptoms
• eyes sensitive to light
• clammy skin.

Your healthcare provider will give you a Patient Safety Card about the risk of serious infections. Carry it with you at all times during treatment and for 2 months after your last dose. Your risk of serious infections may continue for several weeks after your last dose. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

Only available through a restricted program called the Empaveli REMS. Your healthcare provider must be a member of this program to be able to prescribe Empaveli and provide you with the necessary information and Patient Safety Card.

It is not known if Empaveli is safe and effective in children.

Before receiving this medicine

Do not receive Empaveli if you:

• Are allergic to pegcetacoplan, Empaveli, or any inactive ingredients in the injection
• have not been fully vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with Empaveli is needed
• Have an unresolved serious infection caused by encapsulated bacteria.

Before you receive Empaveli, tell your healthcare provider about all of your medical conditions, including if you:

• have an infection or fever
• have not had all your childhood vaccinations
• are pregnant, plan to become pregnant, or are breastfeeding.

Pregnancy

Empaveli may harm your unborn baby. Females who are able to become pregnant should have a pregnancy test before starting treatment and use an effective method of birth control (contraception) during treatment, and for 40 days after the final dose.

Breastfeeding

It is not known if Empaveli passes into breast milk. You should not breastfeed during treatment, and for 40 days after the final dose.

How is Empaveli administered?

Empaveli is given subcutaneously (this means under the skin) using an infusion pump or the Empaveli injector. Use this medicine exactly as your healthcare provider tells you. Your healthcare provider will tell you how much to infuse and how often. Do not infuse more or less than your healthcare provider tells you to.

• Your doctor or nurse will show you how to use the Injector or infusion pump so you can administer it at home, or a caregiver can administer it to you.
• It is usually given twice a week, although a healthcare provider may instruct some people to administer it every three days.
• See the Empaveli Package Insert for more information.

Dosing information

Dose of Empaveli for PNH for adults:

• Recommended dosage: 1080 mg (one injection vial) administered subcutaneously twice weekly.
• Empaveli can be administered via a commercially available infusion pump or with the Empaveli injector. 

Dose of Empaveli for C3G or IC-MPGN 

• Adults 18 and older: 1080 mg (one injection vial) administered subcutaneously twice weekly.
• Children 12 years to less than 18 years: Dosage is based on weight.

Administration via an infusion pump

It should be administered into a clear area of skin on your stomach (avoid around the belly button), or the top of your thighs, hips, or upper arms.

If you are using the infusion pump, it usually takes around 30 minutes to infuse (if using two infusion sites) or approximately 60 minutes (if using one infusion site). See the product information for complete details about the administration process.

Administration via the Empaveli injector

The Empaveli injector is a compact, single-use device that is attached to your body and delivers your dose of pegcetacoplan subcutaneously (under the skin) of your abdomen, allowing you greater mobility while the infusion is in process. A push button starts the injection, and the hidden needle automatically retracts upon dose completion.

What happens if I miss a dose?

If you miss a dose, take the missed dose as soon as possible. Take your next dose at your regularly scheduled time.

Do not stop taking Empaveli without your doctor's advice. After stopping it, your healthcare provider will need to monitor you closely for at least 8 weeks, because stopping treatment may cause a breakdown of red blood cells due to PNH, and symptoms such as:

• decreased hemoglobin levels
• blood in your urine
• shortness of breath
• trouble swallowing
• tiredness
• pain in the stomach (abdomen)
• blood clots
• erectile dysfunction (ED).

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What other drugs will affect Empaveli?

Sometimes it is not safe to use certain medications at the same time as Empaveli. Some drugs can affect your blood levels of other drugs you take, which may increase side effects or make the medications less effective. Tell your doctor about all the vaccinations you have had and what medicines you take, including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements.

Storage

Store vials in the refrigerator between 36°F to 46°F (2°C to 8°C) in the original carton to protect from light.

Do not use past the expiration date stamped on the carton.

Keep all medicines out of the reach of children and pets.

Ingredients

Active ingredient: pegcetacoplan

Inactive ingredients: sorbitol, glacial acetic acid, sodium acetate trihydrate, Water for Injection USP. Empaveli may also contain sodium hydroxide and/or additional glacial acetic acid for pH adjustment.

Injection: 1080 mg/20mL (54 mg/mL) in a single-dose vial.

Manufacturer

Apellis Pharmaceuticals, Inc., makes Empaveli, and their headquarters are in Waltham, MA 02451.

Other notable drugs made by Apellis include Syfovre, an intravitreal formulation of pegcetacoplan administered once monthly into the eye to treat geographic atrophy (GA) caused by age-related macular degeneration (AMD). 

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Further information

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