- Shire's Vonvendi (recombinant von Willebrand factor) has met the primary endpoint in a Phase 3 von Willebrand disease study, controlling bleeding and blood loss during major, minor and oral elective surgery,according to results reported at the annual American Society of Hematology meeting.
- When used before, during or after surgery, bleeding control was rated "excellent" for all 15 treated patients. There were 12 treatment-emergent adverse events (TEAEs) in six people, with two serious TEAEs in two patients, and no inhibitory antibodies.
- Shire hopes to expand the label for Vonvendi, and this study will form part of the evidence that Shire needs for the supplemental NDA in the U.S., as well as for regulatory filings in Europe and elsewhere during 2017.
Vonvendi was the first FDA-approved recombinant von Willebrand factor, which received marketing approval in December 2015 for on demand treatment and control of bleeding episodes in adults with von Willebrand disease. This was followed up by launch in August 2016, after the completion of Shire's purchase of Vonvendi developer Baxalta in June 2016.
"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, head of Research and Development at Shire.
The acquisition of Baxalta means that Shire has gained a portfolio of marketed and pipeline hematology drugs, including drugs in clinical trials for hemophilia and thrombotic thrombocytopenic purpura.
Von Willebrand disease is the most common inherited bleeding disorder, and affects up to 1% of the U.S. population. In these individuals, a deficiency or dysfunction in the von Willebrand factor, a protein, means that their blood does not clot properly. The severity of the disease varies, and can mean anything from easy bruising through to excessive bleeding after surgery and spontaneous bleeds into muscles and joints. Treatments to prevent or treat bleeding include desmopressin, tranexamic acid or von Willebrand factor concentrate purified from donated blood. Shire has plans for pediatric and prophylaxis studies for Vonvendi.