Shire plc, a leading global biotechnology company, announced the US launch of Vonvendi [von Willebrand factor (Recombinant)], the only recombinant treatment for adults living with von Willebrand disease (VWD).

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 3 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, M.D., director, Comprehensive Hemophilia and Thrombosis Center 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 hematology 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 3 multicenter, 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 per cent) reported successful treatment of bleeding episodes, with 96.9 per cent of treated bleeds (N=192 bleeds in 22 patients) achieving an “excellent” efficacy rating and 3.1 per cent achieving a “good” efficacy rating. The most common adverse reaction observed in greater than two percent of patients in clinical trials was generalized pruritus.

Shire is the leading global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. The company strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including hematology, immunology, neuroscience, ophthalmics, lysosomal storage disorders, gastrointestinal/internal medicine/endocrine and hereditary angioedema; and a growing franchise in oncology.