Takeda Recombinant Von Willebrand Factor Phase 3 Clinical Reached Primary Endpoint

On July 21st, Takeda Pharmaceutical announced the results of a Phase 3 clinical trial at the International Society of Thrombosis and Hemostasis (ISTH) 2021 conference
.

The trial was carried out in adult patients with von Willebrand disease (VWD) to test the effectiveness and safety of recombinant von Willebrand factor (rVWF, trade name: Veyvondi/Vonvendi) as a preventive treatment

VWD is the most common hereditary bleeding disorder.
It is caused by von Willebrand factor (VWF) deficiency or dysfunction.
VWF is one of several proteins in the blood that promote normal clotting
.

Due to defects or insufficiency of VWF, the blood of VWD patients cannot be effectively coagulated, leading to prolonged bleeding time

This prospective, open, international multi-center phase 3 study enrolled 23 patients with severe VWD.
The study period was 12 months and included 2 groups: (1) Prior OD group ), this group included patients who had previously received VWF on-demand treatment.
In the study, preventive treatment of rVWF was started, and the infusion was 50±10 IU/kg twice a week; (2) switch group, This group included patients who had previously received plasma-derived von Willebrand factor (pdVWF) prophylactic treatment.
During the study, preventive treatment of rVWF was started, and the dose was matched according to the weekly dose of the previous pdVWF prophylactic treatment regimen (±10%)
.

In the study, a total of 23 patients received rVWF prophylaxis (previous on-demand treatment group: n=13; switching group: n=10), and 18/23 (78.

The results showed that during the 12-month study period: the use of rVWF preventive treatment in the previous on-demand treatment group can effectively reduce the annualized bleeding rate (sABR) of spontaneous and therapeutic bleeding events, and switch from pdVWF preventive treatment to rVWF Patients in the prophylactic treatment switch group maintained the same level of hemostasis control
.

In the study (receiving rVWF preventive treatment), in the previous on-demand treatment group, compared with the patient's historical sABR data, rVWF preventive treatment reduced sABR by 91.

During the 12-month study period, 84.
6% (11/13) patients in the previous on-demand treatment group and 70.
0% (7/10) patients in the switch group had sABR of 0 after treatment
.

Before entering the study, 100% (13/13) of the patients in the previous on-demand treatment group and 10% (1/10) of the patients in the switch group had sABR>2

       In this study, rVWF maintained a favorable benefit risk profile
.

No new risks were discovered, and no serious adverse events related to rVWF were reported

       The use of on-demand treatment can effectively prevent bleeding in patients with VWD, but it cannot prevent later bleeding
.

For many patients treated on-demand, these bleeding complications and unpredictability can affect their daily lives

       Reference source: Takeda data at ISTH 2021 Highlight the Benefits of Prophylaxis for Patients with Rare Bleeding Disorders