Before, I could not make travel plans

Quote from a real EMPAVELI patient.
Image is an actor portrayal.

EMPAVELI was studied vs eculizumab in a head-to-head Phase 3 clinical trial in 80 people living with PNH. Compared to the beginning of the study, people taking EMPAVELI had a 2.37 g/dL increase in hemoglobin levels compared to a 1.47 g/dL decrease with eculizumab at Week 16. EMPAVELI showed a superior 3.84 g/dL difference in Hb levels vs eculizumab (P<0.0001).

Before, I could not make travel plans

Quote from a real EMPAVELI patient.
Image is an actor portrayal.

EMPAVELI was studied vs eculizumab in a head-to-head Phase 3 clinical trial in 80 people living with PNH. Compared to the beginning of the study, people taking EMPAVELI had a 2.37 g/dL increase in hemoglobin levels compared to a 1.47 g/dL decrease with eculizumab at Week 16. EMPAVELI showed a superior 3.84 g/dL difference in Hb levels vs eculizumab (P<0.0001).

How was EMPAVELI studied?

About the study

EMPAVELI was studied in a head-to-head, 16-week, open-label clinical trial open-label clinical trial A type of study in which both the
healthcare professionals and the
patients are aware of the drug or
treatment being given.
compared to eculizumab in 80 people living with PNH.

People in this trial needed to have been on a steady dose of eculizumab for at least 3 months and have low 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 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.
levels (less than 10.5 g/dL) in order to be eligible for the study.

The study consisted of 2 parts:
  • A 4-week period in which people received both EMPAVELI and eculizumab
  • After 4 weeks, people were randomly split into 2 groups, in which they received only EMPAVELI or only eculizumab for 16 weeks
Goals of the study included:
  • Primary goal: to explore changes in Hb levels
  • Secondary goal: to study additional data points
  • Safety: to monitor the occurrence of side effects

See below for more details on the goals and results of the study

Superior improvements in Hb

PRIMARY GOAL: To explore whether EMPAVELI was better at improving Hb levels than eculizumab in people living with PNH.

Compared to the beginning of the study, people taking either treatment had the following at Week 16:

diagram diagram

Additional values studied

KEY SECONDARY GOAL: “Noninferiority” tests were used to study additional information and determine if EMPAVELI was no worse than eculizumab changes in Hb levels. These include:
  • Helping people become transfusion free
  • Changes in reticulocytes (also known as absolute reticulocyte countabsolute reticulocyte count (ARC) A test that measures the number
    of immature red blood cells in your
    blood. High reticulocyte counts may
    be a sign of PNH, as the body is
    making more new red blood cells to
    replace the ones being destroyed by
    the immune system.
    , or ARC)
  • Changes in LDH (also known as lactate dehydrogenase) lactate dehydrogenase (LDH) An enzyme found in the blood
    and tissues of the body, including
    the heart, kidneys, brain, and
    lungs. Red blood cell destruction
    results in the release of LDH into
    the blood. People with PNH often
    have higher levels of LDH.
  • Changes in fatigue

Fatigue was measured using a survey called Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale) FACIT-Fatigue Scale This tool consists of a number of
questions that are used to assign
a score to measure a person’s
experience with fatigue.
, where higher scores on the survey meant a person felt less fatigue.


In this study, EMPAVELI was no worse than eculizumab in achieving transfusion independence and reducing ARC.

At Week 16:

image

Transfusion independence
was achieved for 35 people (85%) taking EMPAVELI who were transfusion free

image
image

Reduced ARC
for people taking EMPAVELI by 136 X 109 cells/L compared to their values at the beginning of the study

EMPAVELI impacts these values that may play a role in PNH
Additional results from the study
LDH was tested for noninferiority, but it could not be determined if EMPAVELI was no worse than eculizumab. Information collected was for observation only and no conclusions can be made on the effect of EMPAVELI on this measure.
  • At Week 16, people taking EMPAVELI had reduced LDH by 15 U/L compared to their LDH values at the beginning of the study
FACIT-Fatigue was not formally tested. Information collected was for observation only and no conclusions can be made on the effect of EMPAVELI on this measure.
  • At Week 16, people taking EMPAVELI had improved fatigue by 9.2 points on the FACIT-Fatigue scale compared to their fatigue levels at the beginning of the study

Normalization of lab values

Hb and ARC normalization were prespecified secondary endpoints,
or goals planned to be evaluated prior to the start of the PEGASUS study

Information collected was for observation only. No conclusions can be made from these data.

In this analysis of prespecified secondary endpoints in the PEGASUS study, at Week 16:

  • Hb normalization was achieved in 34% of people taking EMPAVELI
    Normal Hb is usually between 12 g/dL and 18 g/dL, depending on sex
  • ARC normalization was achieved in 78% of people taking EMPAVELI
    Normal ARC can differ from person to person. It is measured as a range of values, from 30 X 109
    to 120 X 109 cells/L

EMPAVELI was also studied in patients who had never received PNH treatment with a
complement inhibitor before

24 people living with PNH were studied in 2 uncontrolled clinical trials. EMPAVELI was not compared against another treatment. These patients had never received complement treatment before and had at least 1 transfusion in the last year before entering the trial
Patients were on EMPAVELI for about 1 year and increases in hemoglobin were seen
See the possible side effects of treatment
learn more
Image is an actor portrayal.
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 final 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 final 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, viral infection, and tiredness.

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.