Shire, continuing on Baxalta’s longstanding commitment to the bleeding disorder community, has announced the US launch of VONVENDI
As the first and only recombinant von Willebrand factor (VWF) treatment, VONVENDI replaces the VWF the body is missing in VWD. The treatment is also the first in the United States that has the option to dose recombinant von Willebrand factor (rVWF) independent of recombinant Factor VIII (rFVIII), based on patient need. This attribute allows for tailored treatment for patients who may not require additional FVIII.
VWD is a hereditary bleeding disorder that is caused by deficiency or dysfunction of VWF, a plasma protein that mediates the initial adhesion of platelets at sites of vascular injury and also carries and protects factor VIII from premature proteolysis. Because of this, the blood does not clot properly, resulting in heavy menstrual periods, easy bruising or frequent nose bleeds. It is estimated that one in 100 people, approximately three million people in the United States, suffer from VWD.
“As the first and only recombinant treatment option, VONVENDI represents an important step forward in the treatment of VWD,” said Ralph Gruppo, MD, Director, Comprehensive Haemophilia and Thrombosis Centre at the Cincinnati Children’s Hospital. “By being able to replace with only VWF recombinant proteins that function similarly to VWF that occurs naturally in the body, patients and their physicians have an effective new treatment option for this challenging and chronic disease.”
With the availability of VONVENDI, Shire continues to grow its industry leading haematology portfolio, invest in treatment options and support for VWD patients around the world, and aspires to help unite the VWD community to elevate awareness and education. Regulatory submission in Europe is expected in 2017 and in other markets globally. Additionally, VONVENDI is being studied for use in the surgical setting.
“VONVENDI marks a new era in treating von Willebrand disease to help give people with VWD control over their bleeds,” said Shannon Resetich, Head of Global Hematology, Shire. “We look forward to expanding availability to patients who can benefit from this treatment all over the world.”
VONVENDI was approved by the US Food and Drug Administration (FDA) in December 2015 and is indicated for on-demand treatment and control of bleeding episodes in adults (age 18 and older) with VWD. The FDA approval was based on positive results from a Phase III multicentre, open-label clinical trial that assessed the safety, efficacy and pharmacokinetics of VONVENDI with and without recombinant FVIII.
In the pivotal study, all participants (100%) reported successful treatment of bleeding episodes, with 96.9% of treated bleeds (N=192 bleeds in 22 patients) achieving an “excellent” efficacy rating and 3.1% achieving a “good” efficacy rating. The most common adverse reaction observed in greater than 2% of patients in clinical trials was generalized pruritus.