Skip to main content

Table 5 AEs reported by subject incidence (n, %), event rate (events/person-years), and total number of events

From: Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study

Exposure, person-years Parent study (n = 80) Extension study (n = 68) Overall phase 2 treatment (n = 80)a
  25.1   230.0   256.0
Incidence
n (%)
Event rate (total number of events) Incidence
n (%)
Event rate (total number of events) Incidence
n (%)
Event rate (total number of events)
AEs 79 (98.8%) 58.3 (1463) 68 (100%) 18.6 (4284) 80 (100%) 22.5 (5747)
 AEs causing early pegvaliase discontinuation 5 (6.3%) 0.20 (5) 4 (5.9%) 0.05 (12) 9 (11.3%) 0.07 (17)
SAEs 4 (5.0%) 0.16 (4) 11 (16.2%) 0.07 (16) 15 (18.8%) 0.08 (20)
 SAEs causing early pegvaliase discontinuation 1 (1.3%) 0.04 (1) 2 (2.9%) 0.01 (3) 3 (3.8%) 0.02 (4)
Death 0 0 0 0 0 0
AEs of special interest
 Hypersensitivity events 69 (86.3%) 14.9 (373) 62 (91.2%) 4.3 (997) 75 (93.8%) 5.4 (1370)
 Generalized skin reaction (≥14 days) 9 (11.3%) 0.44 (11) 37 (54.4%) 0.40 (93) 39 (48.8%) 0.41 (104)
 Injection-site reaction 68 (85.0%) 19.1 (479) 59 (86.8%) 3.8 (875) 76 (95.0%) 5.3 (1354)
 Injection-site skin reaction (≥14 days) 13 (16.3%) 0.88 (22) 25 (36.8%) 0.27 (62) 36 (45.0%) 0.33 (84)
 Arthralgia 41 (51.3%) 5.6 (140) 34 (50.0%) 0.74 (171) 52 (65.0%) 1.2 (311)
 Acute systemic hypersensitivity event of anaphylaxis 3 (3.8%) 15.9 (4) 1 (1.5%) 0.01 (2) 3 (3.8%) 0.02 (6)
 Acute systemic hypersensitivity event per Brown’s severe criteria 1 (1.3%) 0.04 (1) 1 (1.5%) 0.01 (2) 2 (2.5%) 0.01 (3)
  1. Event rate was calculated as total number of events divided by person-years of exposure
  2. AE adverse event, SAE serious adverse event
  3. aAll phase 2 data are included