Empaveli is a brand-name infusion prescribed for paroxysmal nocturnal hemoglobinuria (a rare blood disorder). Empaveli contains the active ingredient pegcetacoplan and belongs to the complement inhibitors drug class.

Drug details

The following list highlights some key information about Empaveli.

  • Drug class: complement inhibitors
  • Drug form: liquid solution for infusion with an infusion pump or the Empaveli Injector device
  • Generic available?no
  • Prescription required? yes
  • Controlled substance?no
  • Year of FDA approval: 2021

Empaveli has a boxed warning. A boxed warning is the most serious warning from the Food and Drug Administration (FDA). For details, see the “Empaveli precautions” section.

Empaveli is available only as a brand-name medication. It’s not currently available in generic form.

A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics usually cost less than brand-name drugs.

The following information describes dosages that are commonly used or recommended. However, be sure to use the dosage your doctor prescribes for you. Your doctor will determine the best dosage to fit your needs.

Drug forms and strengths

Empaveli comes as a liquid solution in a single-dose vial. The drug is available in one strength: 1,080 milligrams (mg) of the drug in a 20-milliliter (mL) liquid solution.

Your doctor may prescribe Empaveli as an infusion pump or an Empaveli injector. The Empaveli injector is a single-use, wearable device that slowly releases the medication over 30 to 60 minutes.

If you have questions about whether to use Empaveli with an infusion pump or a single-use injector, talk with your doctor. They’ll help determine the best way for you to receive your Empaveli doses.

Dosage for paroxysmal nocturnal hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder in which your immune system destroys red blood cells. This leads to anemia (low red blood cell count).

The recommended dose of Empaveli for PNH is 1,080 mg. You will receive this twice per week as subcutaneous infusions.

Sometimes, blood tests may show that you have a high level of an enzyme called lactate dehydrogenase. If this applies to you, your doctor may change your dosage to 1,080 mg every 3 days.

How to inject Empaveli

You receive Empaveli as a subcutaneous infusion twice per week. This is an injection under the skin that’s given over a period of time using a device known as an infusion pump. A healthcare professional can teach you or your caregiver how to give infusions at home using an infusion pump or a single-use Empaveli injector.

When using an infusion pump, you can inject Empaveli into the following sites:

  • your abdomen, avoiding the area 2 inches (5 centimeters) around your belly button
  • your thighs
  • your hips
  • the back of your upper arms

You should only use the Empaveli injector on the abdomen.

How to use the Empaveli injector

To use an Empaveli injector, the drug’s manufacturer recommends the following:

  1. Take the vial from the refrigerator and let it warm to room temperature for 30 minutes. Do not heat it in any other way, such as using a microwave, hot water, or direct sunlight.
  2. Use a needle-less transfer device to transfer Empaveli from its single-dose vial to the Empaveli injector.
  3. Place the Empaveli injector onto a clean, dry spot on your abdomen. Make sure this injection site is at least 1 inch away from your belly button and any previous injection sites.
  4. Firmly press the activation button to begin the infusion. The activation button will pop back out when the infusion is complete, which can take up to an hour.
  5. Discard the Empaveli injector after one use. Do not reuse the injector device.

Each Empaveli infusion lasts about 30 to 60 minutes. The injector device has a fill gauge that shows you when the infusion is complete.

During your infusion, it’s important to avoid activities that may cause your injector to become wet. This includes bathing, exercise, swimming, and sweating. Excess moisture could limit how well the Empaveli Injector adheres to your skin.

To reduce the risk of injection site reactions, do not inject Empaveli into the exact same spot every time. You should also avoid placing the injector on scars, tattoos, or irritated skin.

For more detailed instructions on using this injector device, talk with your doctor or pharmacist.

What if I miss a dose?

If you miss a dose of Empaveli, you should take it as soon as possible. Then continue with your regular dosing schedule.

Do not take two doses together to make up for a missed dose.

To help make sure that you don’t miss a dose, try using a medication reminder. This can include setting an alarm or timer on your phone or downloading a reminder app.

Will I need to use this drug long term?

Empaveli is meant to be used as a long-term treatment. If you and your doctor determine that Empaveli is safe and effective for you, you’ll likely use it long term.

Empaveli can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while taking Empaveli. These lists do not include all possible side effects.

For more information about the possible side effects of Empaveli, talk with your doctor or pharmacist. They can give you tips on how to manage any side effects that may concern or bother you. You can also refer to Empaveli’s prescribing information.

Note: After the Food and Drug Administration (FDA) approves a drug, it tracks and reviews side effects of the medication. Sharing your experience helps make medicines safer for everyone by giving doctors and researchers more information about how the drug works in real life. If you develop a side effect while taking Empaveli and want to tell the FDA about it, visit MedWatch or call 800-FDA-1088.

Mild side effects

Mild side effects that people receiving Empaveli have reported include:

Most of these side effects may go away within a few days or a couple of weeks. But if they become more severe or don’t go away, talk with your doctor or pharmacist.

For more information about some of these side effects, see the “Side effect details” section.

Serious side effects

Serious side effects from Empaveli aren’t common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency number if your symptoms feel life threatening or if you think you’re having a medical emergency.

Serious side effects of Empaveli can include serious infections, such as pneumonia.

In fact, Empaveli has a boxed warning from the FDA for the risk of serious infections. A boxed warning alerts doctors and patients about drug effects that may be dangerous.

Allergic reaction

For some people, Empaveli can cause an allergic reaction.

But this side effect wasn’t common in the drug’s studies.

In general, symptoms of allergic reaction can be mild or serious.

Ways to manage

For mild allergic reaction symptoms, such as a mild rash, call your doctor right away. They may recommend treatments to help manage your symptoms. They’ll also let you know whether you should keep taking the medication.

For severe allergic reaction symptoms, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms require immediate medical care because they can become life threatening. If you’ve had a serious allergic reaction to Empaveli, your doctor may recommend taking a different medication instead.

Side effect details

Here’s some detail on certain side effects this drug may cause.

Boxed warning: Risk of serious infections

Empaveli has a boxed warning for the risk of serious infections caused by encapsulated bacteria. A boxed warning alerts doctors and patients about drug effects that may be dangerous.

Specifically, Empaveli can weaken part of your immune system. This makes it harder for your body to fight infections caused by encapsulated bacteria.

Examples of encapsulated bacteria include:

  • Streptococcus pneumoniae
  • Neisseria meningitidis types A, B, C, W, and Y
  • Haemophilus influenzae type B

These bacteria can cause serious, life threatening infections such as pneumonia, meningitis, and sepsis (blood infection). Without prompt treatment, these infections can lead to death.

Symptoms of serious infection can include:

What you can do about serious infections

To lower your risk for these infections, make sure you are up to date on your vaccinations. If not, you’ll likely receive a vaccine for the bacteria mentioned before you start treatment with Empaveli. Here’s some information to keep in mind:

  • If you have not received vaccines against these bacteria in the past, you should ideally get them at least 2 weeks before your first dose of Empaveli. This gives the vaccines time to work.
  • If you need urgent treatment with Empaveli, your doctor may want you to start Empaveli sooner than 2 weeks after receiving the vaccines. In this case, you’ll likely also need to take antibiotics to help prevent infections. You’ll take the antibiotics for the first 2 weeks of Empaveli treatment.
  • If you have received vaccines for these bacteria in the past, ask your doctor if you need any booster doses before you start taking Empaveli.

Although vaccination reduces the risk of serious infections, it may not prevent it completely.

Your doctor should give you a patient safety card about the risk of serious infections with Empaveli. Keep this with you while taking Empaveli and for 2 months after your last dose.

If you develop any symptoms of serious infections while taking Empaveli, see your doctor right away. They’ll typically prescribe antibiotics to treat it. You may need to be treated in the hospital. You may also need to stop taking Empaveli until the infection gets better. Call 911 or local emergency number if your symptoms feel life threatening.

Talk with your doctor to learn more about serious infections and Empaveli.

Due to the risk of serious infections, only doctors and people who are enrolled in a certain drug safety program can use Empaveli. The name of the program is the Risk Evaluation and Mitigation Strategy (REMS) program.

In addition to possibly causing the serious infections mentioned above, Empaveli can increase your risk for less serious infections. In fact, infections were among the most common side effects reported in clinical studies of Empaveli.

Examples of these infections include:

Symptoms vary depending on the type of infection. But they may include:

What you can do

To help lower your risk for infections while taking Empaveli, try to avoid close contact with people who are sick or have an infection. You can also follow these tips.

If you have symptoms of an infection while taking Empaveli, talk with your doctor or pharmacist. They may be able to recommend a suitable over-the-counter treatment.

You can also ask your doctor for more information about how to help prevent infections while taking Empaveli.

Injection site reactions

You will receive Empaveli as a subcutaneous infusion. This is an injection under the skin that’s given over a period of time using an infusion pump. It’s possible to develop skin reactions around the area where you receive the infusion. These injection site reactions may include:

  • redness, darkness, or discoloration
  • warmth or swelling
  • bruising
  • hardening
  • itching or pain

In studies, injection site reactions were one of the most common side effects reported with Empaveli. The reactions were typically mild or moderate. Injection site reactions usually go away in a few days.

What you can do

To help reduce your risk for injection site reactions, follow your doctor’s instructions for taking Empaveli.

To help prevent irritation, use a different injection site each time you have an infusion. Make a note of the date and area you use so you can use a different spot the next time. Also, avoid rubbing the injection site after you’ve had your infusion.

Keep in mind that you should not inject Empaveli into areas of skin that are tender, bruised, hard, or discolored. And do not inject the drug into areas with stretch marks, scars, or tattoos.

If you have an injection site reaction, applying a cold pack may help reduce pain or swelling. Tell your doctor if you have an injection site reaction that’s severe or doesn’t go away in a few days.

Digestive problems

Empaveli can sometimes cause digestive problems. These include diarrhea as well as pain, tenderness, or discomfort in your abdomen. In studies, diarrhea and abdominal pain were commonly reported with Empaveli. These side effects are usually mild.

Keep in mind that diarrhea can sometimes be a symptom of an infection in your stomach or intestines, such as stomach flu.

What you can do

If you have diarrhea while taking Empaveli, talk with your doctor or pharmacist. They may recommend taking medication such as loperamide (Imodium). Keep in mind that it’s also important to drink plenty of fluids when you have diarrhea. This helps prevent you from becoming dehydrated.

You should also talk with your doctor if you have abdominal pain that bothers you, so they can help determine if it is due to Empaveli.

The Food and Drug Administration (FDA) approves prescription drugs such as Empaveli to treat certain conditions.

Empaveli for paroxysmal nocturnal hemoglobinuria

Empaveli is FDA approved to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) in adults.

With PNH, your body makes some abnormal red blood cells. They lack a particular protein on their surface that usually protects them from your immune system. As a result, your immune system destroys them.

The destruction of red blood cells is called hemolysis, and a symptom includes red or dark urine. But not everyone with PNH has this symptom. There may be periods when hemolysis worsens, such as when you have an infection, following an accident, or during times of stress.

PNH causes anemia and a low hemoglobin level. (Anemia is a low red blood cell count, and hemoglobin is a red pigment that carries oxygen.) This can cause mild or severe symptoms, depending on how low these levels are. Examples of these symptoms can include:

  • weakness or fatigue
  • tiredness
  • shortness of breath, especially during exercise
  • dizziness or feeling faint
  • high heart rate
  • headache
  • chest pain
  • skin, gums, or nail beds that are paler than usual

Severe hemolysis can also cause other symptoms, such as:

Some people with PNH also have problems with blood clots. These clots may block blood supply to the legs, lungs, liver, stomach, intestines, or brain. Blood clots can cause different symptoms depending on the part of the body they affect.

* Sex and gender exist on spectrums. Use of the term “male” in this article refers to sex assigned at birth.

Effectiveness for paroxysmal nocturnal hemoglobinuria

Empaveli is an effective treatment for PNH. The drug has been shown to increase your hemoglobin level and reduce the need for blood transfusions to treat anemia. To read more about how Empaveli performed in clinical studies, see the drug’s prescribing information.

Empaveli and children

Empaveli is not approved to treat PNH in children. The drug has not been studied in children, so it is unknown if this medication is safe or effective for them.

You’ll usually take Empaveli by itself. But when you first start treatment for paroxysmal nocturnal hemoglobinuria (PNH), you might take Empaveli with other medications.

For example, you may be taking a different drug for PNH, such as eculizumab (Soliris) or ravulizumab (Ultomiris). For information on switching to Empaveli, see the “Common questions about Empaveli” section.

If you have questions about using Empaveli with other drugs, talk with your doctor.

There are no reports of interactions between Empaveli and other medications, supplements, or foods. However, this doesn’t mean that interactions won’t occur.

Before taking Empaveli, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential interactions.

If you have questions about drug interactions that may affect you, ask your doctor or pharmacist.

Empaveli and alcohol

Empaveli is not known to interact with alcohol. But if you have certain side effects from Empaveli, such as diarrhea, headache, or fatigue, drinking alcohol could worsen them.

If you drink alcohol, talk with your doctor about how much is safe to drink while taking Empaveli.

Empaveli and vaccines

Sometimes, your doctor may have you start Empaveli treatment within 2 weeks of receiving certain vaccinations. If this applies to you, you might need to take antibiotics in the first 2 weeks of Empaveli treatment.

The antibiotics help prevent infection while the vaccines start to work. This is important because Empaveli has a boxed warning for the risk of serious infections caused by encapsulated bacteria. To learn more, see the “Empaveli side effects” section.

Empaveli and lab tests

Empaveli may interfere with certain lab tests that measure activated partial thromboplastin time. These tests measure how long it takes blood to clot, which a doctor may refer to as your international normalized ratio (INR). The tests are used to monitor treatment with blood-thinning drugs, such as warfarin (Coumadin, Jantoven). Empaveli can cause incorrect results in these tests.

If you need blood tests to check your clotting time, make sure your doctor knows that you’re taking Empaveli. Your doctor may need to tell the lab to use a different test.

As with all medications, the cost of Empaveli can vary. The actual price you’ll pay can depend on your insurance plan, your location, and the pharmacy you use. It may also depend on whether you receive Empaveli infusions from a healthcare professional or use the Empaveli Injector to self-administer your doses.

Financial and insurance assistance: If you need financial support to pay for Empaveli, or if you need help understanding your insurance coverage, help is available. Empaveli’s manufacturer offers a program called Apellis Assist. For more information and to find out if you’re eligible for support, call 866-MY-APL-ASSIST (866-692-7527). Or you can visit the program website.

Generic version: Empaveli is not available in a generic form. A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics tend to cost less than brand-name drugs.

Learn more about saving money on prescriptions.

Empaveli is used to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH).

With PNH, the immune system destroys red blood cells, leading to anemia (low red blood cell count).

What happens with PNH

Red blood cells develop in your bone marrow (a spongy material inside your bones) from immature cells called stem cells. Usually, red blood cells develop a layer of proteins on their surface that protects them from your immune system. With PNH, a genetic change in certain stem cells causes some red blood cells to develop without this protective layer. These red blood cells are vulnerable to attack from a part of your immune system called the complement system.

The complement system is a group of proteins that work together with other parts of your immune system, including white blood cells and antibodies. The system helps find and destroy foreign or abnormal material in your body.

The complement system finds the abnormal red blood cells in your blood vessels, liver, and spleen. The system destroys these red blood cells through a process called hemolysis, during which the blood cells break apart. When the red blood cells split up, they release hemoglobin (a red pigment that carries oxygen). The hemoglobin leaves your body in your urine and can make your urine red. This is called hemoglobinuria.

People with PNH have anemia. They also have low levels of hemoglobin in their blood. Anemia may need to be treated with blood transfusions.

What Empaveli does

Empaveli works by blocking the complement system in your immune system. The drug does this by attaching to a complement protein known as complement protein C3. This stops the complement system from destroying the red blood cells in your blood vessels, liver, and spleen.

Empaveli helps increase your red blood cell count and hemoglobin level. This helps ease the symptoms of anemia. It can also reduce your need for blood transfusions.

How long does it take to work?

Empaveli starts working soon after your first infusion. It can take a few weeks for the drug to build up its effectiveness, and you notice your symptoms easing.

It is unknown if Empaveli is safe to take during pregnancy. The drug hasn’t been studied in people who are pregnant.

In animal studies, Empaveli increased the risk of pregnancy loss when it was given to pregnant animals. But animal studies don’t always predict what will happen in humans.

If you have paroxysmal nocturnal hemoglobinuria (PNH), it’s usually recommended that you avoid becoming pregnant. PNH can cause serious risks for you and the fetus during pregnancy. These risks include blood clots, infections, bleeding, pregnancy loss, premature delivery, and low levels of blood cells.

If you’re able to become pregnant, your doctor will order a pregnancy test for you before you start treatment with Empaveli. This is so your doctor can be sure that you’re not pregnant before you begin taking the medication.

If you’re pregnant or plan to become pregnant, talk with your doctor about the risks and benefits of taking Empaveli.

It is unknown if Empaveli is safe to take during pregnancy. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control needs while you’re taking Empaveli.

For females taking Empaveli

Females* who could become pregnant should use effective birth control while taking Empaveli and for 40 days after their last dose.

For males taking Empaveli

The manufacturer of Empaveli hasn’t given birth control recommendations for males* taking this drug. If you’re a male and your partner can become pregnant, talk with your doctor about your birth control needs while taking Empaveli.

* Sex and gender exist on spectrums. Use of the term “male” in this article refers to sex assigned at birth.

You should not breastfeed while taking Empaveli or for 40 days after your last dose.

It is unknown if Empaveli passes into breast milk or if it can affect a child who’s breastfed. If Empaveli does pass into breast milk, it could possibly cause serious side effects in a child who is breastfed.

If you’re breastfeeding or planning to, talk with your doctor. They can advise you on alternative ways to feed your child and whether Empaveli is right for you.

Here are answers to some frequently asked questions about Empaveli.

Will I have to be monitored after stopping Empaveli treatment?

Yes, you will need to be monitored after you end Empaveli treatment. After you stop taking Empaveli, your immune system may start to destroy your red blood cells again. This could cause the symptoms of paroxysmal nocturnal hemoglobinuria (PNH) to come back.

Your doctor will monitor your condition for at least 8 weeks after you end Empaveli treatment. You’ll likely have blood tests to check your:

After you stop taking Empaveli, tell your doctor if you have any new or worsening symptoms of PNH. See the “Empaveli uses” section for details.

If your condition worsens after you stop taking Empaveli, your doctor may recommend you restart the treatment. Talk with your doctor if you have other questions about monitoring that you may need.

If needed, how will my doctor switch my treatment from either eculizumab or ravulizumab to Empaveli?

Eculizumab (Soliris) and ravulizumab (Ultomiris) are two other medications that may be used to treat PNH. You may be taking one of these before you start taking Empaveli.

If your doctor has you switch from eculizumab to Empaveli, you’ll likely start taking Empaveli while you’re still using eculizumab. After 4 weeks, you’ll stop using eculizumab and continue to use Empaveli alone.

If your doctor has you switch from ravulizumab to Empaveli, you’ll likely start taking Empaveli within 4 weeks of stopping ravulizumab treatment. Talk with your doctor if you have any questions about switching or which PNH treatment may be right for you.

The timing involved in switching drugs helps prevent a condition called hemolysis, which is the destruction of red blood cells.

Does Empaveli cure paroxysmal nocturnal hemoglobinuria?

No, Empaveli doesn’t cure PNH. The drug works to stop your immune system from destroying your abnormal red blood cells. Empaveli also helps correct anemia and low hemoglobin level. But the medication doesn’t stop your bone marrow (the spongy tissue inside your bones) from making abnormal red blood cells. If you stop treatment with Empaveli, your immune system will typically start to destroy your abnormal red blood cells again.

The only cure for PNH is to have a bone marrow transplant.

To find out more, talk with your doctor. They can tell you what to expect from taking Empaveli. They can also advise you on whether you could be a candidate for a bone marrow transplant.

This drug comes with several precautions.

FDA warning: Serious infections

This drug has a boxed warning. This is the most serious warning from the Food and Drug Administration (FDA). A boxed warning alerts doctors and patients about drug effects that may be dangerous.

Empaveli may increase your risk for serious infections caused by certain bacteria, especially a type called encapsulated bacteria. The infections can include pneumonia, meningitis, and sepsis (blood infection). If not treated quickly, these infections can, in rare cases, lead to death.

To reduce your risk for these infections, you should be up to date on your vaccinations. If not, you’ll likely need to be vaccinated against the bacteria before you start treatment with Empaveli.

If you have not had these vaccines in the past, you should ideally get them at least 2 weeks before your first dose of Empaveli. This gives the vaccines time to work. If your doctor wants you to start Empaveli sooner than this, you’ll also need to take antibiotics for the first 2 weeks of Empaveli treatment. If you have had vaccines against these bacteria in the past, ask your doctor if you need any booster doses before you start Empaveli.

Although vaccination reduces the risk of these serious infections, it may not prevent them completely. See your doctor right away if you have symptoms of serious infections while taking Empaveli, such as:

Due to the risk of serious infections, only doctors who are enrolled in a certain drug safety program can prescribe Empaveli. The name of the program is the Risk Evaluation and Mitigation Strategy (REMS) program.

Other precautions

Before taking Empaveli, talk with your doctor about your health history. Empaveli may not be right for you if you have certain medical conditions or other factors affecting your health. These include:

  • Allergic reaction: You shoud not take Empaveli if If you have had an allergic reaction to Empaveli or any of its ingredients. Ask your doctor what other medications are better options for you.
  • Pregnancy: It is unknown if it is safe to receive Empaveli during pregnancy. For more information, see the “Empaveli and pregnancy” section.
  • Breastfeeding: You shouldn’t breastfeed while taking Empaveli or for 40 days after stopping treatment. For more information, see the “Empaveli and breastfeeding” section.

Note: For more information about the potential negative effects of Empaveli, see the “Empaveli side effects” section.

Disclaimer: Medical News Today has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.