PNH

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When PNH is uncontrolled,
your symptoms don’t stop

Actor portrayal.

Treating your PNH means focusing on what causes PNH

Paroxysmal nocturnal hemoglobinuria, or PNH, is a rare blood disease that causes your immune systemimmune systemA sophisticated defense network used to protect the body from dangers like disease and infection. to attack and destroy your red blood cells in a process called hemolysishemolysisThe destruction of red blood cells by the complement system, which is part of your immune system.. Red blood cells carry an important molecule called hemoglobin throughout your body.

PNH develops when there is a change in a part of the body’s DNA, which is called a genetic mutation. Because of this mutation, red blood cells are missing critical proteins responsible for protecting them from attack.

There are 2 types of hemolysis:


intravascular hemolysis (IVH)intravascular hemolysis (IVH)Veins, capillaries, and arteries are types of blood vessels inside your body. IVH occurs when red blood cells are attacked and destroyed inside of the blood vessels.

which occurs in the blood vessels


extravascular hemolysis (EVH)extravascular hemolysis (EVH)The spleen and liver are 2 organs in your body that act like recycling centers for your red blood cells. EVH occurs when red blood cells are attacked and destroyed in these organs.

which occurs in the liver or spleen

Hemolysis leads to fewer red blood cells and less hemoglobin (Hb)hemoglobin (Hb)The critical protein found inside red blood cells that enables other cells throughout the body to get the oxygen they need.Hemoglobin binds to oxygen in the lungs and carries it to other tissues. It can also transport waste like carbon dioxide to help remove it from the body. to carry oxygen in your blood.

This may cause your PNH symptoms.

PNH symptoms can disrupt your life, and may not stop with treatment

Whether you have been newly diagnosed or are currently being treated with a therapy like a C5 inhibitorC5 inhibitorA treatment that targets the C5 protein in the complement system (part of the immune system). C5 is a protein located in the blood that plays an important role in inflammation and intravascular hemolysis caused by the complement system., you could be experiencing some of the signs and symptoms below. If you are, you may want to discuss treatment options with your doctor to address these ongoing signs and symptoms.

Headaches

Fatigue, or feeling weak or tired

Fatigue is a common symptom of PNH and can be associated with both IVH and EVH. If your treatment only targets IVH, you can be left with persistent anemia due to EVH. Such anemia contributes to fatigue that may make doing everyday tasks and activities difficult.

Trouble concentrating(often called “brain fog”)

Difficulty exercising

Trouble breathing

Loss of appetite

AnemiaanemiaA condition due to the body not having enough healthy red blood cells, which carry oxygen throughout the body.
Hemolysis, both IVH and EVH, can cause anemia as your red blood cells are attacked and destroyed. Only treating IVH may still lead to persistent anemia as EVH can continue to cause low red blood cell counts. You may need blood transfusions to manage it.

Bruising or bleeding easily

Small red dots on the skin

A rapid heartbeat

Blood in urine

Blood clots
(approximately 15%-30% of people with PNH develop blood clots, especially in the veins)

Abdominal and chest pain

Actor portrayal.

PNH symptoms can disrupt your life, and may not stop with treatment

Whether you have been newly diagnosed or are currently being treated with a therapy like a C5 inhibitorC5 inhibitorA treatment that targets the C5 protein in the complement system (part of the immune system). C5 is a protein located in the blood that plays an important role in inflammation and intravascular hemolysis caused by the complement system., you could be experiencing some of the symptoms below. If you are, you may want to discuss treatment options with your doctor to address these ongoing symptoms.

Actor portrayal.

Headaches

Fatigue, or feeling weak or tired

Fatigue is a common symptom of PNH and can be associated with both IVH and EVH. If your treatment only targets IVH, you can be left with persistent anemia due to EVH. Such anemia contributes to fatigue that may make doing everyday tasks and activities difficult.

Trouble concentrating(often called “brain fog”)

Difficulty exercising

Trouble breathing

Loss of appetite

AnemiaanemiaA condition due to the body not having enough healthy red blood cells, which carry oxygen throughout the body.
Hemolysis, both IVH and EVH, can cause anemia as your red blood cells are attacked and destroyed. Only treating IVH may still lead to persistent anemia as EVH can continue to cause low red blood cell counts. You may need blood transfusions to manage it.

Bruising or bleeding easily

Small red dots on the skin

A rapid heartbeat

Blood in urine

Blood clots
(approximately 15%-30% of people with PNH develop blood clots, especially in the veins)

Abdominal and chest pain

This list includes common signs and symptoms of PNH and is not comprehensive. This list is for educational purposes, and symptoms can vary for each person. EMPAVELI is approved to treat PNH, not specific symptoms.
Talk to your doctor about any symptoms you are experiencing.

If you are on a C5 inhibitor and you continue to have symptoms,
your PNH may not be fully controlled

C5 inhibitors only work to stop IVH, so you may still experience symptoms related to EVH. These symptoms, like persistent anemia and fatigue, can affect your daily life and have health and personal consequences.

Treatments that control both types of hemolysis may be more effective for managing ongoing symptoms.

If symptoms like fatigue impact your daily life despite treatment, talk to your doctor about an option designed to control both kinds of hemolysis

Discover what you can achieve

EVH=extravascular hemolysis; Hb=hemoglobin; IVH=intravascular hemolysis; 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.