Table 2.
PK parameters
| Cohort 1 (50 IU/kg efanesoctocog alfa [n = 9]) | Cohort 2 (65 IU/kg efanesoctocog alfa [n = 14]) | |||
|---|---|---|---|---|
| Day 1* | Day 22 | Day 1* | Day 22 | |
| t1/2, h | — | 41.3 (37.0-46.1) | — | 37.3 (34.6-40.2) |
| Cmax, IU/dL† | 113 (98-130) | 131 (110-157) | 158 (144-174) | 171 (157-186) |
| AUC0-τ, h × IU/dL | 7650 (6750-8670) | 8290 (7260-9460) | 10 500 (9 230-12 000) | 11 200 (9 800-12 800) |
| CLss, mL/h/kg | — | 0.60 (0.53-0.69) | — | 0.58 (0.51-0.66) |
| IR, IU/dL per IU/kg | 2.26 (1.95-2.61) | 2.43 (2.03-2.92) | 2.43 (2.21-2.68) | 2.45 (2.26-2.66) |
| MRTinf, h | — | 62.3 (56.2-69.1) | — | 58.9 (55.7-62.2) |
| Accumulation index‡ | — | 1.07 (1.05-1.09) | — | 1.05 (1.04-1.06) |
CLss, clearance at steady state; Cmax, maximum FVIII activity; IR, incremental recovery; MRT, mean residence time.
All data are geometric mean (95% confidence interval) and are based on the 1-stage activated partial thromboplastin time clotting assay.
*
Some parameters were not calculated for the first dose administered because they were more accurately calculated after the final dose when blood sampling could continue for 14 days postdose.
†
Day 22 value is Cmax at steady-state.
‡
Accumulation index was defined as the ratio of AUC0-τ at steady-state/AUC0-τ after the first dose.