“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.
Individual experiences may vary.
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.
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
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
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
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
At Week 16, EMPAVELI was no worse than a C5i (eculizumab) in helping people with PNH avoid transfusions and reducing ARC
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
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
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
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)
- 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.
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