NEJM:BIVV001 maintains long-term high levels of VIII factor activity in patients with severe type A haemophilia.

VIII factor substitutes greatly improve the condition of patients with type A haemophilia, but most substitutes have a shorter half-life, greatly limiting their clinical application, although the half-life of recombinant VIII factor products can be increased to 15-19 hours, but more effective methods to improve the half-life of VIII factors are still needed.
BIVV001 (rFVIIIFc-VWF-XTEN) is a new fusion protein, and researchers recently examined the safety and pharmacodynamic data of single-dose BIVV001 therapy.
study was conducted in Phase 1-2a. Sixty-six men (18-65 years old) with severe type A haemophilia (factor VIII activity -lt;1%) received a single intravenous recombinant VIII factor at a dose of 25 IU (low dose group) or 65 IU/kg (high dose group) per kilogram of body weight, at least 3 days after injection, and then the patient received a single intravenous BIVV001 dose of 25 IU or 65 IU per kilogram.
of the study were adverse events and pharmacodynamic data.
28 days after the injection of BIVV001 structure and single dose BIVV001 of the dosing scheme, no inhibition of VIII factors was detected and no allergic events occurred.
the geometric average half-life of BIVV001 is 3 to 4 times that of recombined VIII factors (low dose group: 37.6 vs 9.1 hours, high dose group: 42.5 vs 13.2 hours).
area (AUC) under the VIII factor curve of the two BIVV001 dose groups is 6 to 7 times that of the recombined VIII factor (low dose group: 4470 vs 638 hours x IU per liter, high dose group: 12800 vs 1960 hours x IU per liter).
injection of high-dose BIVV001, the average level of VIII factors in patients on day 4 reached the normal range (-51%), and on the 7th day it was 17%, indicating that BIVV001 therapy could achieve weekly interval medication.
difference in VIII factor activity between groups of patients with severe type A haemophilia, single intravenous BIVV001 can maintain a high level of VIII factor activity for a long time, half-life is 4 times the recombinant VIII factor, and good safety.
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