Table 2.
Safety overview in patients with PH1 during lumasiran treatment
| Event, n (%) | Placebo/lumasiran (n = 13) | Lumasiran/lumasiran (n = 26) | All lumasiran (N = 39) |
|---|---|---|---|
| Any AE | 9 (69) | 24 (92) | 33 (85) |
| Serious AEa | 0 | 1 (4) | 1 (3) |
| Severe AEa | 0 | 1 (4) | 1 (3) |
| AE leading to discontinuation of study treatmentb | 0 | 1 (4) | 1 (3) |
| AEs occurring in ≥10% of patients | |||
| Injection-site reactionsc | 5 (39) | 11 (42) | 16 (41) |
| Abdominal pain | 1 (8) | 6 (23) | 7 (18) |
| Headache | 0 | 4 (15) | 4 (10) |
| Rhinitis | 2 (15) | 2 (8) | 4 (10) |
| Upper respiratory tract infection | 1 (8) | 3 (12) | 4 (10) |
| Death | 0 | 0 | 0 |
AE, adverse event; PH1, primary hyperoxaluria type 1.
Safety data from first dose of lumasiran to data cutoff date of May 1, 2020.
a
Urosepsis, considered not related to study drug by the investigator.
b
Fatigue and disturbance in attention, considered not related to lumasiran by the investigator.
c
Includes AEs of injection-site reaction, injection-site pain, injection-site erythema, and injection-site discomfort.