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THIS SITE IS INTENDED FOR US RESIDENTS 18 YEARS AND OLDER.
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This site is intended for US residents 18 years and older
Are you still experiencing PNH symptoms?
They may be the result of ongoing hemolysis

Why hemolysis matters

What is hemolysis?

Hemolysis hemolysis The destruction of red blood
cells by the complement system. is the destruction of red blood cells red blood cells A common type of blood cell.
Their job is to carry oxygen,
using an important molecule,
hemoglobin, around the body. by the complement system complement system The complement system, also referred to
as the complement cascade, is an army of
more than 40 different proteins that work
together to get rid of dangerous cells and
fight infections. .

Despite improvements while on treatment, some people living with paroxysmal nocturnal hemoglobinuria paroxysmal nocturnal hemoglobinuria PNH is a blood disease in which the
immune system—our body’s natural
defense system—attacks and destroys red
blood cells. (PNH) still experience:

  • Fatigue
  • Abnormal lab values

These may be signs that your red blood cells are being destroyed in the body.

There are 2 types of hemolysis in PNH

Most treatments only help with 1 kind of hemolysis,
known as intravascular hemolysis.

What are the 2 types of hemolysis?
Intravascular hemolysis (IVH)
is when red blood cells are
destroyed inside the blood vessels.
Extravascular hemolysis (EVH)
is when red blood cells are destroyed outside
the blood vessels, in the liver and spleen.
Both types of hemolysis may be
causing PNH symptoms and low hemoglobin

The vital role played by hemoglobin

Hemoglobin in healthy circulation

Hemoglobin Hemoglobin (Hb) The critical protein found inside red blood
cells that enables other cells throughout
the body to get the oxygen they need.
Hemoglobin acts like drops of glue that can
“stick” to oxygen and carry it from the lungs
to other tissues. It can also “stick” to waste
like carbon dioxide to help remove it from
the body. is the critical protein found inside red blood cells that enables other cells and organs throughout the body to get the oxygen they need.

Hemoglobin levels fall in PNH

Ongoing destruction of red blood cells means not enough hemoglobin is available to carry out efficient gas exchange as you breathe.

Low hemoglobin levels lead to anemia, which can cause symptoms like fatigue, headaches, and trouble breathing

Other factors to consider in PNH

Absolute reticulocyte count (ARC)

ARC measures the number of immature red blood cells. A very high number of these cells may be a sign that the body is trying to make up for too few mature red blood cells and low hemoglobin levels.

Transfusions

Needing blood transfusions a few times a year may be a sign of inadequate hemolytic control.

Fatigue

Fatigue is a common symptom for people with PNH because low hemoglobin levels mean that not enough oxygen can circulate through the body.

Lactate dehydrogenase (LDH)

LDH is an enzyme involved in energy production, abundant in red blood cells. Levels above normal may indicate that those cells are continuing to rupture.

The goal of treatment is to improve these factors
See how EMPAVELI works
learn more
Jillian is an adult with PNH taking EMPAVELI.
INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION AND IMPORTANT SAFETY INFORMATION

What is EMPAVELI® (pegcetacoplan)?

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

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

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

EMPAVELI may increase your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.

EMPAVELI may also increase the risk of getting serious infections caused by certain bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Serious infections may quickly become life-threatening and cause death if not recognized and treated early.

  1. You must be vaccinated against these bacteria at least 2 weeks before your first dose of EMPAVELI if you have not already had these vaccines.
  2. If your healthcare provider decides that urgent treatment with EMPAVELI is needed, you should receive the required vaccinations as soon as possible.
  3. If you have not been vaccinated and EMPAVELI therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
  4. If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting EMPAVELI. Your healthcare provider will decide if you need additional vaccinations.
  5. 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:
    • 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
    • clammy skin
    • 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. It is important to show this card to any healthcare provider to 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, give you a Patient Safety Card about your risk of serious infections, and make sure that you are vaccinated.

Who should NOT take EMPAVELI?

Do not take EMPAVELI if you:

  • are allergic to pegcetacoplan or any of the ingredients in EMPAVELI.
  • have not been vaccinated against Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B, unless your healthcare provider decides that urgent treatment with EMPAVELI is needed.
  • have a serious infection caused by the bacteria above.

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
  • swelling of your face, tongue, or throat
  • feel faint or pass out

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.

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