Shire has announced topline results from a Phase 3 clinical trial of Vonvenditm [von Willebrand factor (Recombinant)] to treat bleeds in elective surgical settings for adults with severe von Willebrand disease (VWD), the most common inherited bleeding disorder.
Vonvendi is an on-demand recombinant treatment for adults living with von Willebrand disease 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 von Willebrand disease.
von Willebrand disease is the most common inherited bleeding disorder, affecting up to 1 percent of the U.S. population.
Shire said that 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 .
“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,” said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. “Shire is committed to advancing research and possible care options for patients with von Willebrand disease, building on our commitment to improve outcomes for patients with a range of bleeding disorders.”
Shire announced presentation of the complete results of the study 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, Shire said.