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“My hemoglobin was below normal and now it is comfortably above normal, which is what we were hoping for.”

EMPAVELI was studied vs eculizumab in a Phase 3 trial in 80 adults with PNH. Compared to the beginning of the study, people taking EMPAVELI had a 2.37 g/dL increase in hemoglobin (Hb) 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). Patients who continued on EMPAVELI were able to maintain higher Hb levels through 48 weeks.

PNH=paroxysmal nocturnal hemoglobinuria.

Louis is an adult with PNH taking EMPAVELI.
Individual experiences may vary.

EMPAVELI efficacy

EMPAVELI was studied in adults with previous C5i treatment (eculizumab) and those without any previous complement inhibitor treatment. View the EMPAVELI results from the PEGASUS study and from the PRINCE study below.

PEGASUS Study
PRINCE Study

Results at Week 16: Superior improvements in Hb

Primary goal: to explore whether EMPAVELI was better at improving Hb levels than a C5i (eculizumab) in people living with PNH

Average change in Hb levels from beginning to Week 16

SE=standard error.

Patients who continued on EMPAVELI were able to maintain higher Hb levels through 48 weeks

About the PEGASUS study

EMPAVELI was compared to eculizumab in 80 adults with PNH.

People in this study needed to have been on a steady dose of eculizumab for at least 3 months and have low 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 3 parts:

  • For the first 4 weeks, people received both EMPAVELI and eculizumab
  • Then, people were randomly split into 2 groups for 16 weeks:
    • Some received only EMPAVELI
    • Some received only eculizumab
  • Then, people from both groups received only EMPAVELI for 32 more weeks
    • Patients in the eculizumab group received both eculizumab and EMPAVELI for 4 weeks before transitioning to just EMPAVELI

ecu=eculizumab; EMP=EMPAVELI.

Goals of the study included:

  • Primary goal: to explore changes in Hb levels
  • Secondary goal: to study additional factors that can impact PNH
  • Safety: to monitor the occurrence of side effects

Key secondary goals

The PEGASUS study also looked at:

  • Seeing how many people with PNH could become transfusion-free
  • Changes in absolute reticulocyte count (ARC) absolute 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.
  • Changes in lactate dehydrogenase (LDH) 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

Noninferiority tests were used to study these measures and determine if EMPAVELI was no worse than eculizumab compared to the beginning of the study.

Key secondary results: avoiding transfusions, reducing ARC

Key secondary results

At Week 16, EMPAVELI was no worse than a C5i (eculizumab) in helping people with PNH avoid transfusions and reducing ARC

Avoiding transfusions

Avoiding transfusions

85% of people taking EMPAVELI were transfusion-free

15% of people taking a C5i (eculizumab) were transfusion-free

Average changes in ARC

Average changes in ARC

People taking EMPAVELI saw their ARC reduced by 136 x 109 cells/L

People with PNH taking a C5i (eculizumab) saw an increase in ARC of 28 x 109 cells/L

Additional secondary results: measuring LDH and fatigue

Additional results

LDH measures

LDH measures

For this measure, it could not be determined if EMPAVELI was any worse than a C5i (eculizumab)

  • At Week 16, people taking EMPAVELI had reduced LDH by 15 U/L compared to their LDH values at the beginning of the study
  • People taking a C5i (eculizumab) saw their LDH levels drop by 10 U/L compared to their values at the beginning of the study
  • This information was for observation only and no conclusions can be made on the effect of EMPAVELI on LDH levels

Fatigue

Fatigue

Although it was not formally tested, people in the study were asked to report their levels of fatigue

  • Fatigue was measured using a survey called Functional Assessment of Chronic Illness Therapy-Fatigue (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.     . Higher scores on the survey meant a person felt less fatigue
  • At Week 16, people taking EMPAVELI had an improvement of 9.2 points on the FACIT-Fatigue scale compared to their fatigue levels at the beginning of the study
  • People taking a C5i (eculizumab) had a decrease of -2.7 points
  • Report of fatigue may be an under- or overestimation since patients were aware of the treatment they were being given
  • This information was for observation only and no conclusions can be made on the effect of EMPAVELI on fatigue

Post hoc analysis of PEGASUS data assessing Hb and fatigue

Change in FACIT-Fatigue by Hb at Week 16 (N=72)

Patients with greater improvements in Hb showed larger reductions in fatigue
(nominal* P<0.0001)
  • Higher FACIT-Fatigue scores indicate lower levels of fatigue
  • Post hoc analyses gather additional findings after a study has ended. They are not considered as scientifically strong as preplanned analyses
  • This post hoc analysis included patients receiving EMPAVELI and eculizumab

*Nominal P value signifies that the analysis was planned only after the study had ended.

See side effects in PEGASUS study

With EMPAVELI, Hb was stabilized and LDH decreased

% of people who achieved Hb stabilization

Most people in the EMPAVELI group achieved Hb stabilization through Week 26 vs no people in the control arm (excluding C5i).

Decreases in LDH levels observed through 26 weeks†

By Week 2, average LDH levels in the EMPAVELI group had dropped from baseline and remained there through 26 weeks.

*Hb stabilization means avoiding more than a 1 g/dL drop in Hb levels throughout the study.

†Control arm (excluding C5i) only includes data on or before escape (switching to EMPAVELI).

About the PRINCE study

EMPAVELI was compared to a control arm, which included people on stable doses of medications and treatments for their PNH (excluding C5is like eculizumab or ravulizumab)

53 adults with PNH were randomly divided into 2 groups and studied for 26 weeks.
Group 1 received EMPAVELI and Group 2 (the control arm) continued their PNH treatment without a C5i or EMPAVELI. These people had never received complement treatment for their PNH before entering the trial.

Most people in the trial had been getting frequent blood transfusions to make up for low Hb levels. People entering the PRINCE study had received an average of 4 transfusions in the preceding year.

Goals of the study included:

  • Primary goal 1: to stabilize Hb levels*
  • Primary goal 2: to observe changes in LDH levels

See side effects in PRINCE study

See the possible side effects of treatment
learn more
Theresa 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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