Date: 2016-12-02
Type of information: Results
phase: 3
Announcement: results
Company: Shire (UK - USA)
Product: Vonvendi®(recombinant von Willebrand Factor)
Action
mechanism: protein. Vonvendi® (recombinant von Willebrand Factor) is an on-demand recombinant treatment for adults living with von Willebrand disease and replaces von Willebrand factor (VWF).
Disease: von Willebrand disease
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial details:
Latest
news: * On December 2, 2016, Shire announced topline results from a Phase 3 clinical trial of Vonvendi® (von Willebrand factor) to treat bleeds in elective surgical settings for adults with severe von Willebrand disease. 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 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 be used to support the filing for regulatory approvals in Europe in 2017. They will also form the basis of a supplemental new drug application to the FDA, of which Shire is requesting for an expanded indication for Vonvendi®.
