Table 22Summary of Harms

Adverse eventsALXN1210-PNH-301ALXN1210-PNH-302
Ravulizumab

N = 125

Eculizumab

N = 121

Ravulizumab

N = 97

Eculizumab

N = 98

Patients with ≥ 1 AE
n (%)110 (88.0)105 (86.8)85 (87.6)86 (87.8)
Most common eventsaNANANANA
    Headache45 (36.0)40 (33.1)26 (26.8)17 (17.3)
    Upper respiratory tract infection13 (10.4)7 (5.8)18 (18.6)10 (10.2)
    Nausea11 (8.8)10 (8.3)8 (8.2)9 (9.2)
    Nasopharyngitis11 (8.8)18 (14.9)21 (21.6)20 (20.4)
    Diarrhea10 (8.0)5 (4.1)9 (9.3)7 (7.1)
    Viral upper respiratory tract infection9 (7.2)10 (8.3)1 (1.0)0 (0.0)
    Pain in extremity9 (7.2)7 (5.8)5 (5.2)4 (4.1)
    Dizziness9 (7.2)7 (5.8)3 (3.1)7 (7.1)
    Arthralgia8 (6.4)8 (6.6)3 (3.1)4 (4.1)
    Oropharyngeal pain8 (6.4)6 (5.0)4 (4.1)9 (9.2)
    Abdominal pain7 (5.6)7 (5.8)6 (6.2)9 (9.2)
    Myalgia7 (5.6)9 (7.4)2 (2.1)4 (4.1)
    Back pain7 (5.6)6 (5.0)4 (4.1)4 (4.1)
    Pyrexia6 (4.8)13 (10.7)9 (9.3)5 (5.1)
    Hypokalemia6 (4.8)6 (5.0)0 (0.0)1 (1.0)
    Vomiting5 (4.0)4 (3.3)6 (6.2)4 (4.1)
    Fatigue5 (4.0)4 (3.3)6 (6.2)6 (6.1)
    Dyspepsia4 (3.2)6 (5.0)0 (0.0)2 (2.0)
    Musculoskeletal pain4 (3.2)2 (1.7)2 (2.1)5 (5.1)
    Cough4 (3.2)8 (6.6)5 (5.2)10 (10.2)
    Anemia3 (2.4)5 (4.1)6 (6.2)3 (3.1)
    Influenza-like illness3 (2.4)1 (0.8)7 (7.2)8 (8.2)
    Dyspnea3 (2.4)2 (1.7)0 (0.0)6 (6.1)
    Chest pain2 (1.6)5 (4.1)3 (3.1)9 (9.2)
    Rhinitis2 (1.6)3 (2.5)5 (5.2)4 (4.1)
    Insomnia2 (1.6)6 (5.0)1 (1.0)1 (1.0)
    Constipation1 (0.8)3 (2.5)7 (7.2)5 (5.1)
    Infusion-related reactionb1 (0.8)0 (0.0)3 (3.1)1 (1.0)
Patients with ≥ 1 SAE
n (%)11 (8.8)9 (7.4)4 (4.1)8 (8.2)
Most common eventscNANANANA
    Hemolysis0 (0.0)0 (0.0)0 (0.0)2 (2.0)
    Pyrexia1 (0.8)2 (1.7)0 (0.0)3 (3.1)
Patients who stopped treatment due to AEs
n (%)0 (0.0)0 (0.0)0 (0.0)0 (0.0)
Deaths
n (%)0 (0.0)1 (0.8)d0 (0.0)0 (0.0)
Patients with notable AEs, n (%)
Serious infections2 (1.6)4 (3.3)2 (2.1)1 (1.0)
Infusion reactions11 (8.8)10 (8.3)8 (8.2)3 (3.1)
Patients with notable SAEs, n (%)
Infections and infestationsNANANANA
  Abscess limb0 (0.0)1 (0.8)0 (0.0)0 (0.0)
  Cellulitis0 (0.0)1 (0.8)0 (0.0)0 (0.0)
  Infection0 (0.0)1 (0.8)0 (0.0)0 (0.0)
  Influenza0 (0.0)0 (0.0)1 (1.0)0 (0.0)
  Leptospirosis1 (0.8)0 (0.0)0 (0.0)0 (0.0)
  Lower respiratory tract infection0 (0.0)0 (0.0)1 (1.0)0 (0.0)
  Pneumonia0 (0.0)1 (0.8)0 (0.0)0 (0.0)
  Pyelonephritis acute0 (0.0)0 (0.0)0 (0.0)1 (1.0)
  Systemic infection1 (0.8)0 (0.0)0 (0.0)0 (0.0)
  Viral upper respiratory tract infection0 (0.0)1 (0.8)0 (0.0)0 (0.0)

AE = adverse event; NA = not applicable; SAE = serious adverse event.

Note: AEs are reported for the safety set.

a

Frequency of 5% or greater in any treatment group.

b

Identified as a notable harm in the systematic review protocol.

c

Frequency of greater than 1% in any treatment group.

d

Death due to lung adenocarcinoma occurred in the extension phase.

Source: Clinical Study Reports for Stud 301 and Study 302.1,2

From: Clinical Review

Cover of Ravulizumab (Ultomiris)
Ravulizumab (Ultomiris): CADTH Reimbursement Reviews and Recommendations: Therapeutic area: Paroxysmal nocturnal hemoglobinuria [Internet].
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