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news.A phase 3 clinical trial of Shire's VONVENDI [von Willebrand factor (Recombinant)] to treat bleeds in elective surgical settings for adults with severe von Willebrand disease (VWD) has met primary endpoint.
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VONVENDI is an on-demand recombinant treatment for adults living with VWD and replaces von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting missing in patients with VWD.
The study met its primary endpoint and indicates that VONVENDI effectively controlled bleeding and blood loss during an operation in adults undergoing major, minor and oral elective surgical procedures. The results will form the basis of a supplemental new drug application to the U.S. Food & Drug Administration, of which Shire is requesting for an expanded indication for VONVENDI.
Shire Research and Development head Philip J. Vickers, Ph.D. said: "These findings suggest that using VONVENDI to treat bleeds during surgery may offer this patient population a new option designed to help meet individual patient needs during surgery.
"Shire is committed to advancing research and possible care options for patients with VWD, building on our commitment to improve outcomes for patients with a range of bleeding disorders."
The prospective, uncontrolled, open-label, nonrandomized, international, multi-center Phase 3 study enrolled 15 patients 18 years and older diagnosed with severe congenital VWD undergoing major and minor elective surgical procedures.
The study assessed the safety and hemostatic efficacy of VONVENDI used before, during or after a surgical procedure, with or without, a recombinant Factor VIII (rFVIII) treatment. Overall hemostatic efficacy was rated as "excellent" or "good" for all 15 treated subjects.
A total of 12 treatment emergent adverse events (TEAEs) occurred in six patients during or after infusion treatment. Two serious TEAEs occurred in two patients. Ten non-serious TEAEs occurred in six patients. None of the TEAEs that occurred during the study were assessed as severe allergic reaction.
In addition, none of the study subjects developed inhibitory antibodies to VONVENDI, rFVIII or binding antibodies to CHO, rFurin or murine IgG.
The complete results of the study will be presented at an upcoming medical conference. The results will also be used to support the filing for regulatory approvals in Europe in 2017 and in other markets globally. VONVENDI is also being studied for prophylaxis and pediatric indications.