TABLE 1.
Steady‐state pegvaliase PK parameters in PRISM‐2, weeks 5–6 of maintenance phase (part 3)
| Parameter | Statistic | Stayed on active treatment | Switched from placebo | ||
|---|---|---|---|---|---|
|
20 mg/day n = 20 |
40 mg/day n = 18 |
20 mg/day n = 11 |
40 mg/day n = 8 |
||
| AUC0–24 (ng/hr/ml) | n | 17 | 12 | 7 | 7 |
| Mean (SD) | 262,181.5 (280,378.2) | 246,782.5 (338,587.5) | 116,564.8 (111,571.1) | 454,920.8 (649,658.7) | |
| Range | 5470–1,126,030 | 4507–1,169,209 | 10,583–319,763 | 5482–1,425,093 | |
| Cmax (ng/ml) | n | 17 | 15 | 8 | 8 |
| Mean (SD) | 14,042.6 (16,254.7) | 16,687.3 (19,457.1) | 5334.5 (5209.9) | 23,053.8 (35,974.3) | |
| Range | 255–68,500 | 239–63,800 | 536–15,500 | 91–91,500 | |
| Ctrough (ng/ml) | n | 15 | 14 | 8 | 8 |
| Mean (SD) | 11,177.3 (8974.7) | 10,446.0 (12,723.9) | 4995.9 (5216.1) | 17,408.5 (29,014.3) | |
| Range | 210–29,600 | 176–43,100 | 347–15,500 | 0–66,100 | |
| Tmax (hour) | n | 17 | 15 | 8 | 8 |
| Mean (SD) | 9.8 (8.1) | 7.5 (4.6) | 16.6 (7.4) | 8.4 (7.8) | |
| Range | 0–24 | 0–12 | 8–24 | 0–24 | |
| t1/2 (hour) | n | 13 | 8 | 5 | 3 |
| Mean (SD) | 47.3 (41.6) | 60.2 (44.6) | 49.6 (37.9) | 26.5 (11.9) | |
| Range | 14–132 | 14–127 | 15–115 | 14–37 | |
Abbreviations: AUC0–24, area under the cure from 0 to 24 hours; Cmax, maximum concentration; Ctrough, trough blood pegvaliase concentrations; PK, pharmacokinetic; SD, standard deviation; t1/2, time to 50% reduction in Cmax; Tmax, time to reach Cmax.