Empaveli

Empaveli (pegcetacoplan) is a complement inhibitor used to treat:

• adults with paroxysmal nocturnal hemoglobinuria (PNH)
• adults and children 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria (protein in the urine) and the loss of kidney function.

Empaveli binds to complement protein C3 and its activation fragment C3b. These are key parts of the complement system, a part of the immune system that, when overactive, can destroy red blood cells in PNH or damage the kidneys in C3G and IC-MPGN.

Empaveli is given as a subcutaneous (under the skin) infusion twice a week using an infusion pump or the Empaveli Injector, a single-use on-body injector. It can be given at home after training from a healthcare provider.

Empaveli was first approved by the FDA on May 14, 2021, for PNH. It was approved for C3G and primary IC-MPGN on July 28, 2025, and the label was updated in August 2026 to include reducing the loss of kidney function. There is no generic.

See the detailed Instructions for Use that comes with your Empaveli for information about how to prepare and infuse your dose of Empaveli with your infusion pump or with your Empaveli Injector.

• Your healthcare provider should show you how to prepare and administer Empaveli before you use it for the first time.
• Use Empaveli exactly as your healthcare provider tells you. Do not use more or less than your healthcare provider tells you to.
• Take the vial out of the refrigerator and let it reach room temperature for about 30 minutes before use. Keep it in the carton until you are ready to use it.
• Empaveli should be clear and colorless to slightly yellowish. Do not use it if it looks cloudy, contains particles, or is dark yellow.
• With an infusion pump, rotate infusion sites between the abdomen, thighs, hips, and upper arms. If you use 2 sites, keep them at least 3 inches apart. An infusion takes about 30 minutes using 2 sites or about 60 minutes using 1 site.
• The Empaveli Injector is only for use on the abdomen. An injection takes about 30 to 60 minutes.
• Do not infuse into skin that is tender, bruised, red, or hard, or into tattoos, scars, or stretch marks. Throw away any unused medicine.

What is an Empaveli Injector?

The Empaveli Injector is a compact, single-use, wearable device that attaches to your body and delivers your prescribed dose of Empaveli subcutaneously (under the skin) into your abdomen.

The device features a hidden needle that automatically retracts when the dose is complete, and a simple push-button mechanism to start the injection.

For adults with PNH

• Empaveli is given under the skin (subcutaneously) 2 times each week. If your LDH level (an enzyme in your blood) rises to more than 2 times the upper limit of normal, your healthcare provider may change your dose to every 3 days and check your LDH twice a week for at least 4 weeks.
• If you are changing treatment from eculizumab to Empaveli, you should continue eculizumab for 4 weeks after your first dose of Empaveli. After 4 weeks, you should stop treatment with eculizumab.
• If you are changing treatment from ravulizumab to Empaveli, you should take your starting dose of Empaveli no more than 4 weeks after your last dose of ravulizumab.

If you have PNH and you stop taking Empaveli, your healthcare provider will need to monitor you closely for at least 8 weeks after stopping Empaveli. Stopping treatment with Empaveli may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include:

• Decreased hemoglobin level in your blood
• Blood in your urine
• Shortness of breath
• Tiredness
• Pain in the stomach (abdomen)
• Blood clots
• Trouble swallowing
• Erectile dysfunction (ED).

For adults and children 12 years and older with C3G or primary IC-MPGN

Empaveli is given under the skin (subcutaneously) 2 times each week. For children 12 to less than 18 years of age, the dose is based on body weight.

What happens if I miss a dose?

If you miss a dose of Empaveli, administer the missed dose as soon as possible. Administer your next dose at your regularly scheduled time.

Empaveli is a medicine that affects your immune system and may lower the ability of your immune system to fight infections. Empaveli carries a Boxed Warning for serious infections caused by encapsulated bacteria.

Serious infections caused by encapsulated bacteria

Empaveli increases your chance of getting serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or cause death if not recognized and treated early.

• You must complete, or be up to date with, vaccines against S. pneumoniae and N. meningitidis (serogroups A, C, W, Y, and B) at least 2 weeks before your first dose of Empaveli.
• If you have not completed your vaccines and Empaveli must be started right away, you should receive the required vaccines as soon as possible.
• If you have not been vaccinated and Empaveli must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you.
• If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting Empaveli. You may also need to be revaccinated during treatment. Your healthcare provider will decide if you need additional vaccines.

Vaccines do not prevent all infections caused by encapsulated bacteria. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious infection:

• Fever with or without shivers or the chills, with chest pain and cough, with a high heart rate, with a rash, or with breathlessness or fast breathing
• Headache with fever, with nausea or vomiting, or with a stiff neck or stiff back
• Confusion
• Clammy skin
• Body aches with flu-like symptoms
• Eyes sensitive to light.

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 of Empaveli. Your risk of serious infections may continue for several weeks after your last dose of Empaveli. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

Empaveli REMS

Empaveli is only available through a program called the Empaveli Risk Evaluation and Mitigation Strategy (Empaveli REMS). Before you can take Empaveli, your healthcare provider must:

• Enroll in the Empaveli REMS program
• Counsel you about the risk of serious infections caused by certain bacteria
• Give you information about the symptoms of serious infections
• Make sure that you are vaccinated against serious infections caused by encapsulated bacteria, and that you receive antibiotics if you need to start Empaveli right away and you are not up to date on your vaccines
• Give you a Patient Safety Card about your risk of serious infections.

Only pharmacies certified in the Empaveli REMS can dispense Empaveli. For more information, visit www.empavelirems.com or call 1-888-343-7073.

Do not receive Empaveli if you:

• Are allergic to pegcetacoplan, Empaveli, or any of the inactive ingredients in the injection (see the end of this guide for a list of ingredients)
• Have a serious infection caused by encapsulated bacteria, including S. pneumoniae, N. meningitidis, or H. influenzae type B, when you are starting Empaveli treatment.

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

• Have an infection or fever
• Are pregnant or plan to become pregnant
• Are breastfeeding or plan to breastfeed.

It is not known if Empaveli is safe and effective in children with PNH, or safe and effective in children under 12 years of age with C3G or primary IC-MPGN.

Pregnancy

Empaveli may harm your unborn baby. Females who can become pregnant:

• Should have a pregnancy test before starting treatment with Empaveli
• Should use an effective method of birth control (contraception) during treatment and for 40 days after the last dose.

Breastfeeding

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

Common side effects

The most common side effects in adults with PNH treated with Empaveli are:

• Injection-site reactions, such as redness, pain, or swelling at the injection site
• Infections
• Diarrhea
• Pain in the stomach (abdomen)
• Respiratory tract infections
• Pain in the arms, hands, legs, or feet
• Low potassium levels in the blood
• Tiredness
• Viral infection
• Cough
• Joint pain
• Dizziness
• Headache
• A rash.

The most common side effects in adults and children 12 years and older with C3G or primary IC-MPGN treated with Empaveli are:

• Injection-site reactions
• Fever
• Common cold
• Flu
• Cough
• Nausea.

How common are Empaveli side effects?

Injection-site reactions in PNH studies were mild or moderate. Less common but clinically relevant reactions reported in fewer than 5% of people with PNH included intestinal ischemia (reduced blood flow to the bowel), biliary sepsis, and hypersensitivity pneumonitis (lung inflammation).

Serious side effects

See Important information above for the risk of serious infections caused by encapsulated bacteria. Empaveli may also cause the following other serious side effects:

Allergic reactions. Allergic reactions, including anaphylaxis, can happen during your Empaveli infusion and can be life-threatening. Stop your infusion and get emergency medical care right away if you get any of these symptoms during your Empaveli infusion:

• Chest pain
• Trouble breathing or shortness of breath
• Wheezing
• Swelling of your face, tongue, or throat
• Feeling dizzy, faint, or like you might pass out
• Fast heart rate
• Nausea or vomiting
• Feeling confused or anxious
• Skin reactions, including rash, hives, and itching.

Tell your healthcare provider about any side effect that bothers you or that does 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.

Adult dose of Empaveli for PNH

1,080 mg subcutaneously twice weekly. The dose may be changed to 1,080 mg every 3 days if LDH is more than 2 times the upper limit of normal.

Dose of Empaveli for C3G or primary IC-MPGN

Adults 18 years and older: 1,080 mg (20 mL) subcutaneously twice weekly.

Children 12 to less than 18 years: dosage is based on body weight.

Empaveli (pegcetacoplan) binds to complement protein C3 and its activation fragment C3b. This regulates the breakdown of C3 and reduces the activity of the complement cascade further downstream.

In PNH, red blood cells are destroyed in 2 ways: inside blood vessels (intravascular hemolysis) and outside blood vessels, mainly in the liver and spleen (extravascular hemolysis). Because Empaveli acts early (proximally) in the complement cascade, it controls both types.

In C3G and primary IC-MPGN, overactive complement causes C3 fragments to build up in the kidney's filtering units (glomeruli). Empaveli reduces this C3 buildup. In the VALIANT study, 74% of people on Empaveli with evaluable kidney biopsies had a large decrease in C3 staining at 26 weeks, compared with 12% on placebo.

Empaveli belongs to the drug class called complement inhibitors.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The prescribing information for Empaveli does not list any specific drug interactions.

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

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

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

Keep all medicines out of the reach of children.

Active ingredient: pegcetacoplan

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

Injection: 1,080 mg/20 mL (54 mg/mL) in a single-dose vial.