Pronunciation: em-PAH-vee-li
Generic name: pegcetacoplan
Dosage form: injection, for subcutaneous use, via an infusion pump or Empaveli injector
Drug class: Selective immunosuppressants

Medically reviewed by Carmen Pope, BPharm. Last updated on Oct 12, 2023.

What is Empaveli?

Empaveli (pegcetacoplan) is an immunosuppressant that is given by subcutaneous (under the skin) infusion, which may be used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare acquired, life-threatening disease of the blood that is characterized by the destruction of red blood cells (hemolytic anemia), blood clots (thrombosis), and impaired bone marrow function.

Empaveli works by binding to specific proteins in the complement system, which is 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 to stop intravascular hemolysis (IVH or the destruction of red blood cells in the circulation) and extravascular hemolysis (EVH or 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.

Empaveli was FDA approved on May 14, 2021 and is only approved to treat PNH.

Warnings

Empaveli can lower the ability of your immune system to fight infections and increase 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. Seek immediate medical attention if you develop any of the following, difficulty breathing including shortness of breath and wheezing, swollen tongue or throat, feeling faint, rapid heart rate, skin reactions, including hives and itching, nausea or vomiting, confusion and anxiety, dizziness or fainting.

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]).

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 taking this medicine

Before you take 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.

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.

Pregnancy and breastfeeding

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.

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

Who should not take Empaveli?

Do not take Empaveli if you:

• are allergic to pegcetacoplan or any of the other 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.

How should I take Empaveli?

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.

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 level in your blood
• 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 are the side effects of Empaveli?

Allergic reactions can happen during the administration of Empaveli. 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:

• 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 include:

• 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.

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 FDA at 1-800-FDA-1088 or to Apellis Pharmaceuticals, Inc at 1-833-866-3346.

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.

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