Clinical Trials

Date: 2015-04-17

Type of information: Presentation of results at a congress

phase: 3

Announcement: presentation of results at the 2015 Scientific Symposium of the Hemostasis and Thrombosis Research Society (HTRS) in New Orleans,

Company: Baxter (USA - IL)

Product: Vonvendi®/Veyvondi®- BAX111 (recombinant von Willebrand Factor)

Action mechanism: protein.

Disease: Von Willebrand Disease

Therapeutic area: Hematological diseases - Genetic diseases - Rare diseases

Country:

Trial details:

Latest news:

• • On April 17, 2015, Baxter International presented additional data from the Phase III clinical trial of BAX 111, the first highly-purified recombinant von Willebrand Factor (rVWF) in clinical development as a treatment for patients with von Willebrand disease, the most common type of inherited bleeding disorder. The data were presented as an Abstract of Distinction during an oral session at the 2015 Scientific Symposium of the Hemostasis and Thrombosis Research Society (HTRS) in New Orleans, and expand on the topline data first disclosed in 2014. The trial met its primary efficacy endpoint defined by the number of patients who achieved treatment success for control of bleeding episodes.
• All patients treated in the full analysis set (N=22) experienced a 100 percent treatment success rating based on a 4-point efficacy rating scale, comparing estimated number of infusions needed to treat the bleeding episodes to the actual number of infusions administered. Efficacy for all treated bleeds (N=192) was rated excellent (96.9%) or good (3.1%), including major bleeds (6 excellent and 1 good). The median number of infusions required to treat bleeding events in the trial was 1 and the majority of events (81.8%) were resolved with a single infusion.
• The multi-center, open-label clinical trial was designed to assess the safety, efficacy and pharmacokinetics of BAX 111 among patients with severe von Willebrand disease aged 18 to 65 years. Bleeding events that occurred during the study were treated with rVWF (40-60 IU/kg; up to 80 IU/kg for major bleeds) initially together with rFVIII and subsequently alone if hemostatic FVIII levels were maintained. rVWF pharmacokinetic (PK) parameters were determined with and without rFVIII and repeated after 6 months for rVWF; these analyses found that rVWF PK was not affected by administration together with rFVIII. The recombinant technology used to produce BAX 111 avoids certain protein maturation processes that typically occur with plasma fractionation. This preserves large functional units known as ultra large multimers, which have been shown to be efficient in clot formation and FVIII stabilization. No patients developed inhibitors or binding antibodies to the treatment, and there were no reports of thrombotic events or severe allergic reactions. Eight adverse events (AEs) were considered causally related to BAX 111: six non-serious related AEs occurred in four patients, and two related serious AEs (chest discomfort and increased heart rate) occurred in one patient.
• The data support the application Baxter submitted to the FDA in late 2014 for approval of BAX 111. Both the European Commission and the FDA have granted orphan-drug designation for BAX 111.

Is general: Yes