Table 3.
AEs that occurred during the 26-week RCP
| Overall AEs and severity, n (%) | Overall pegcetacoplan includes escape patients∗ (n = 46) | Control group, supportive care only† (n = 18) |
|---|---|---|
| Any AE | 33 (71.7) | 12 (66.7) |
| AEs considered related to pegcetacoplan | 13 (28.3) | NA |
| SAE | 4 (8.7) | 3 (16.7) |
| Related to pegcetacoplan | 0 | NA |
| AEs based on severity | ||
| Mild | 16 (34.8) | 7 (38.9) |
| Moderate | 13 (28.3) | 3 (16.7) |
| Severe | 4 (8.7) | 2 (11.1) |
| AEs occurring in ≥5% of pegcetacoplan-treated patients | ||
| Hypokalemia | 6 (13.0) | 2 (11.1) |
| Dizziness | 5 (10.9) | 0 |
| Fever/pyrexia | 4 (8.7) | 0 |
| Ecchymosis | 3 (6.5) | 0 |
| Arthralgia | 3 (6.5) | 0 |
| Headache | 3 (6.5) | 0 |
| AEs of special interest‡ | ||
| Injection site reactions§ | 14 (30.4) | NA |
| Infections‖ | 9 (19.6) | 5 (27.8) |
| Sepsis | 0 | 0 |
| Hypersensitivity¶ | 9 (19.6) | 1 (5.6) |
| Hemolytic disorders | 0 | 0 |
| Thrombosis | 0 | 0 |
NA, not applicable.
Patients randomly assigned to the control group could escape to the pegcetacoplan group if their hemoglobin levels decreased ≥2 g/dL below their baseline measurement or had a qualifying thromboembolic event secondary to PNH.
Control group patients received supportive care (eg, transfusions, corticosteroids, and supplements [iron, folate, and vitamin B12]).
AEs of special interest are either relevant to PNH or to the mechanism of action or route of administration of pegcetacoplan.
Injection site reactions included bruising, hemorrhage, induration, inflammation, rash, peripheral swelling, puncture site reaction, vaccination site reaction, ecchymosis, erythema, and hematoma.
Infections included viral infection, arthritis reactive, COVID-19, COVID-19 pneumonia, cellulitis, nasopharyngitis, pharyngitis, tuberculosis, upper respiratory tract infection, urinary tract infection enterococcal, vaginal infection, influenza, Pneumocystis jirovecii pneumonia, and urinary tract infection.
Hypersensitivity was defined as erythema, rash, allergic cough, dermatitis, dermatitis contact, eyelid edema, injection site rash, rash maculopapular, and rhinitis allergic.