studied vs
a C5i (eculizumab) in the
PEGASUS trial1
The PEGASUS trial (N=80)
was a Phase 3, randomized 1:1,
open-label, head-to-head,
16-week trial of EMPAVELI vs
eculizumab in eculizumab-treated
patients with PNH1
Head-to-head vs C5i (eculizumab)1

PEGASUS consisted of1:

  • A baseline timepoint, in which clinical measurements were taken while patients were on their current stable dose of eculizumab for ≥3 months
  • A 4-week run-in period, in which all patients continued their current dose of eculizumab with the addition of twice-weekly EMPAVELI
  • An RCP, in which patients were randomized 1:1 on Day 1 to monotherapy with EMPAVELI or eculizumab for 16 weeks
  • A 32-week OLP following completion of the study, in which patients received monotherapy with EMPAVELI
Peg is a real EMPAVELI patient.
Select inclusion criteria
Baseline characteristics
Adults ≥18 years with PNH stable dose of eculizumab for ≥3 months1
Low hemoglobin
(Hb levels <10.5 g/dL)1
Patient baseline demographics and characteristics in
Age (years) Mean (SD) 50.2 (16.3) 47.3 (15.8)
Female n (%) 27 (65.9) 22 (56.4)
Asian n (%) 5 (12.2) 7 (17.9)
Black or African American n (%) 2 (4.9) 0
White n (%) 24 (58.5) 25 (64.1)
Other n (%) 0 1 (2.6)
Not reported n (%) 10 (24.4) 6 (15.4)
Hispanic or Latino n (%) 2 (4.9) 1 (2.6)
Not Hispanic or Latino n (%) 29 (70.7) 32 (82.1)
Not reported n (%) 10 (24.4) 6 (15.4)
Hemoglobin level (g/dL)
Mean (SD) 8.7 (1.1) 8.7 (0.9)
Absolute reticulocyte count (X 109/L)
Mean (SD) 218.7 (75.0) 216 (69.1)
LDH level (U/L)
Mean (SD) 257.5 (97.7) 308.6 (284.8)
Number of transfusions in last 12 months prior to Day 28
Mean (SD) 6.1 (7.3) 6.9 (7.7)
<4 n (%) 20 (48.8) 16 (41.0)
≥4 n (%) 21 (51.2) 23 (59.0)
Patients included in PEGASUS had varying transfusion history
(<4 or ≥4 transfusions in prior year)1
Phase 3, head-to-head PEGASUS trial
The run-in period helped minimize the risk of hemolysis while patients started on therapy with EMPAVELI
people group image
  • A total of 38 patients in the group treated with EMPAVELI and 39 patients in the eculizumab group completed the 16-week RCP and continued into the 32-week OLP1
  • All patients who completed the 48-week period were eligible to enroll in a separate long-term extension study. Patients who did not enter the long-term extension were followed for 12 additional weeks1
Trial endpoints
Primary endpoint
Change in Hb levels from baseline to Week 16 (during RCP) vs
Key secondary endpoints
Transfusion avoidance, change in ARC, LDH, and FACIT-Fatigue score from baseline to Week 16 vs
secondary endpoints
Normalization of Hb and ARC3

Additional Analyses
Normalization of

checklist image
Discover the PEGASUS data
See results
ARC=absolute reticulocyte count; C5i=complement component 5 inhibitor; FACIT-Fatigue=Functional Assessment of Chronic Illness Therapy; Hb=hemoglobin; LDH=lactate dehydrogenase; OLP=open-label period; PNH=paroxysmal nocturnal hemoglobinuria; RCP=randomized control period.

References: 1. EMPAVELI [prescribing information]. Waltham, MA: Apellis Pharmaceuticals, Inc.; 2021. 2. Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384:1028-1037. 3. Data on file. Apellis Pharmaceuticals, Inc., Waltham, MA.

Indication and Important Safety Information


EMPAVELI® (pegcetacoplan) is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).


EMPAVELI® (pegcetacoplan) is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA Meningococcal infections may occur in patients treated with EMPAVELI and may become rapidly life-threatening or fatal if not recognized and treated early. Use of EMPAVELI may predispose individuals to serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B.

  • Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria.
  • Vaccinate patients at least 2 weeks prior to administering the first dose of EMPAVELI unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection.
  • Vaccination reduces, but does not eliminate, the risk of serious infections. Monitor patients for early signs of serious infections and evaluate immediately if infection is suspected.

EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Under the EMPAVELI REMS, prescribers must enroll in the program.


  • Hypersensitivity to pegcetacoplan or to any of the excipients
  • Not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
  • Unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae


Serious Infections Caused by Encapsulated Bacteria

The use of EMPAVELI may predispose individuals to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib). To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with EMPAVELI.

For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI. If immediate therapy with EMPAVELI is indicated, administer required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis.

Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider discontinuation of EMPAVELI in patients who are undergoing treatment for serious infections.


Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program and must counsel patients about the risk of serious infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated against encapsulated bacteria. Enrollment and additional information are available by telephone: 1-888-343-7073 or at www.empavelirems.com.

Infusion-Related Reactions

Systemic hypersensitivity reactions (e.g., facial swelling, rash, urticaria) have occurred in patients treated with EMPAVELI. One patient (less than 1% in clinical studies) experienced a serious allergic reaction which resolved after treatment with antihistamines. If a severe hypersensitivity reaction (including anaphylaxis) occurs, discontinue EMPAVELI infusion immediately, institute appropriate treatment, per standard of care, and monitor until signs and symptoms are resolved.

Monitoring PNH Manifestations after Discontinuation of EMPAVELI

After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction. Monitor any patient who discontinues EMPAVELI for at least 8 weeks to detect hemolysis and other reactions. If hemolysis, including elevated LDH, occurs after discontinuation of EMPAVELI, consider restarting treatment with EMPAVELI.

Interference with Laboratory Tests

There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.


The most common adverse reactions (incidence ≥10% of patients) with EMPAVELI vs. eculizumab were injection-site reactions (39% v. 5%), infections (29% v. 26%), diarrhea (22% v. 3%), abdominal pain (20% v. 10%), respiratory tract infection (15% v. 13%), viral infection (12% v. 8%), and fatigue (12% v. 23%).


Females of Reproductive Potential

EMPAVELI may cause embryo-fetal harm when administered to pregnant women. Pregnancy testing is recommended for females of reproductive potential prior to treatment with EMPAVELI. Advise female patients of reproductive potential to use effective contraception during treatment with EMPAVELI and for 40 days after the last dose.

Please see full Prescribing Information, including Boxed WARNING regarding serious infections caused by encapsulated bacteria, and Medication Guide.