PNH

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Understanding
EMPAVELI safety

Laura is an adult with PNH who’s taken EMPAVELI

Most common side effects in the EMPAVELI studies, PEGASUS and PRINCE

The safety of EMPAVELI has been studied in multiple clinical trials.

In people with previous
C5 inhibitor treatment 
In people without previous
complement inhibitor treatment
Long-term safety

Most common side effects (≥5%) in patients treated with EMPAVELI

in the PEGASUS study vs eculizumab

The following reactions were mild or moderate in severity: diarrhea, fatigue, fever,
viral infections, and injection site reactions

  • Serious adverse reactions were reported in 17% of patients treated with EMPAVELI
    • The most common serious adverse reaction was infections (5%)
  • Breakthrough hemolysis occurred in 10% of those taking EMPAVELI vs 23% of those taking eculizumab at Week 16 in the PEGASUS study. Three people taking EMPAVELI left the study due to hemolysis
  • Two people taking EMPAVELI during the additional 32 weeks of the PEGASUS study had side effects related to blood clots. They were deemed not related to EMPAVELI

*Grouped terms.
†Term includes injection-site erythema, injection-site reaction, injection-site swelling, injection-site induration, injection-site bruising, injection-site pain, injection-site pruritus, vaccination site reaction, administration site swelling, injection-site hemorrhage, injection-site edema, injection-site warmth, administration site pain, application site pain, injection-site mass, injection-site rash, vaccination site pain.

Most common side effects (≥5%) in patients treated with EMPAVELI

in the PRINCE study vs control

Injection site reactions and infections were mild or moderate in severity

  • One patient treated with EMPAVELI died due to septic shock
  • Serious adverse reactions were reported in 13% of patients treated with EMPAVELI
  • No patients experienced side effects related to blood clots during the PRINCE study

EMPAVELI (N=46) group includes patients who received EMPAVELI at any point during the study, including patients randomized to EMPAVELI (n=35) and patients randomized to the control arm and crossed over to EMPAVELI treatment (n=11).
*Control arm=supportive care (excluding complement inhibitors).
†Grouped terms.
‡Term includes injection-site bruising, injection-site hemorrhage, injection-site swelling, application site reaction, infusion-site pruritus, injection-site erythema, injection-site rash, puncture site reaction.

Up to 3 years of safety data from an integrated analysis

Most common side effects

  • The most common adverse events (≥10%)* were nasopharyngitis (16.7%), upper respiratory tract infection (14.4%), urinary tract infection (12.9%), injection site reactions (36.4%), abdominal pain (18.2%), fatigue (18.2%), headache (16.7%), and cough (12.1%)—consistent with those reported in the original studies

Serious side effects

  • Two serious adverse events deemed related to EMPAVELI occurred: 1 case of biliary sepsis (severe infection of the gallbladder and associated structures) in PEGASUS and 1 case of sepsis (severe, potentially life-threatening infection throughout the body) in PRINCE
  • During the long-term integrated analysis, 1 person taking EMPAVELI had side effects related to blood clots. They were deemed not related to EMPAVELI
  • Overall, no new side effects were identified

*COVID-19 was excluded due to the pandemic and underlying immunocompromised population of PNH.

  • There have been no cases of encapsulated meningococcal infections in any adults with PNH treated with pegcetacoplan, as of 8/13/2025. Patients were vaccinated prior to treatment with pegcetacoplan

These are not all the possible side effects of EMPAVELI. Be sure to tell your healthcare provider about any side effect that bothers you or that does not go away. Side effects can be reported to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as 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.

Thinking about EMPAVELI as a treatment option for you?

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PNH=paroxysmal nocturnal hemoglobinuria.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about EMPAVELI?

EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as 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.

  1. You must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of EMPAVELI.
  2. If you have not completed your vaccines and EMPAVELI must be started right away, you should receive the required vaccines as soon as possible.
  3. 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.
  4. If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccines.
  5. 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
    • fever with chest pain and cough
    • fever with high heart rate
    • headache and a fever
    • confusion
    • clammy skin
    • fever and a rash
    • fever with breathlessness or fast breathing
    • headache with nausea or vomiting
    • headache with a stiff neck or stiff back
    • 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 EMPAVELI dose. 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 is only available through a program called the EMPAVELI Risk Evaluation and Mitigation Strategy (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, and give you a Patient Safety Card about your risk of serious infections.

Who should NOT take EMPAVELI?

Do not take EMPAVELI if you:

  • are allergic to pegcetacoplan or any of the ingredients in EMPAVELI.
  • have a serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B when you are starting EMPAVELI treatment.

Before you take 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. EMPAVELI may harm your unborn baby. Females who are able to become pregnant should have a pregnancy test before starting treatment with EMPAVELI and use an effective method of birth control during treatment with EMPAVELI and for 40 days after the last dose.
  • are breastfeeding or plan to breastfeed. 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.

Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

If 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
  • trouble swallowing
  • tiredness
  • pain in the stomach (abdomen)
  • blood clots
  • erectile dysfunction (ED)

What are the possible side effects of EMPAVELI?

EMPAVELI can cause serious side effects including allergic reactions. Allergic reactions can happen during your EMPAVELI infusion. Stop your EMPAVELI infusion and tell your healthcare provider or 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
  • feel dizzy or faint or pass out
  • fast heart rate
  • nausea or vomiting
  • feel confused or anxious
  • skin reactions, including rash, hives, and itching

The most common side effects in people with PNH treated with EMPAVELI include injection-site reactions; infections; diarrhea; pain in the stomach (abdomen); respiratory tract infection; pain in the arms, hands, legs, or feet; low potassium in blood; tiredness; viral infection; cough; joint pain; dizziness; headache; and rash.

These are not all of the possible side effects of EMPAVELI. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

What is EMPAVELI® (pegcetacoplan)?

EMPAVELI is a prescription medicine used to treat adults with a disease called paroxysmal nocturnal hemoglobinuria (PNH).

Please see full Prescribing Information, including Boxed WARNING regarding risk of serious infections, and Medication Guide for additional information.

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IMPORTANT SAFETY INFORMATION

What is the most important information I should know about EMPAVELI?

EMPAVELI is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

EMPAVELI increases your chance of getting serious infections caused by encapsulated bacteria such as 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.

  1. You must complete or be up to date with the vaccines against Streptococcus pneumoniae and Neisseria meningitidis at least 2 weeks before your first dose of EMPAVELI.
  2. If you have not completed your vaccines and EMPAVELI must be started right away, you should receive the required vaccines as soon as possible.
  3. 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.
  4. If you have been vaccinated against these bacteria in the past, you might need additional vaccines before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccines.
  5. 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
    • fever with chest pain and cough
    • fever with high heart rate
    • headache and a fever
    • confusion
    • clammy skin
    • fever and a rash
    • fever with breathlessness or fast breathing
    • headache with nausea or vomiting
    • headache with a stiff neck or stiff back
    • 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 EMPAVELI dose. 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 is only available through a program called the EMPAVELI Risk Evaluation and Mitigation Strategy (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, and give you a Patient Safety Card about your risk of serious infections.

Who should NOT take EMPAVELI?

Do not take EMPAVELI if you:

  • are allergic to pegcetacoplan or any of the ingredients in EMPAVELI.
  • have a serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B when you are starting EMPAVELI treatment.

Before you take 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. EMPAVELI may harm your unborn baby. Females who are able to become pregnant should have a pregnancy test before starting treatment with EMPAVELI and use an effective method of birth control during treatment with EMPAVELI and for 40 days after the last dose.
  • are breastfeeding or plan to breastfeed. 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.

Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

If 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
  • trouble swallowing
  • tiredness
  • pain in the stomach (abdomen)
  • blood clots
  • erectile dysfunction (ED)

What are the possible side effects of EMPAVELI?

EMPAVELI can cause serious side effects including allergic reactions. Allergic reactions can happen during your EMPAVELI infusion. Stop your EMPAVELI infusion and tell your healthcare provider or 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
  • feel dizzy or faint or pass out
  • fast heart rate
  • nausea or vomiting
  • feel confused or anxious
  • skin reactions, including rash, hives, and itching

The most common side effects in people with PNH treated with EMPAVELI include injection-site reactions; infections; diarrhea; pain in the stomach (abdomen); respiratory tract infection; pain in the arms, hands, legs, or feet; low potassium in blood; tiredness; viral infection; cough; joint pain; dizziness; headache; and rash.

These are not all of the possible side effects of EMPAVELI. Tell your healthcare provider about any side effect that bothers you or that does not go away.

Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

What is EMPAVELI® (pegcetacoplan)?

EMPAVELI is a prescription medicine used to treat adults with a disease called paroxysmal nocturnal hemoglobinuria (PNH).

Please see full Prescribing Information, including Boxed WARNING regarding risk of serious infections, and Medication Guide for additional information.