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9 Aug 2016

Vonvendi, the first and only recombinant treatment for adults affected by von Willebrand disease, launches in the US

Vonvendi is a significant advancement for the treatment of adults with von Willebrand disease.

Shire, continuing on Baxalta’s long-standing commitment to the bleeding disorder community, has 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 US 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 US, 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, 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 VWD 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.”

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