Rare Daily Staff
The European Commission granted Shire marketing authorization for Veyvondi as a treatment for von Willebrand disease, a rare, genetic bleeding disorder.
VWD is the most common inherited bleeding disorder, affecting up to 1 percent of the global population. It is caused by a deficiency or dysfunction of VWF, one of several types of proteins in the blood that are needed to facilitate proper blood clotting. The mutation may occur spontaneously with no previous family history. Only a minor proportion of affected individuals have the severe form of the disease and need VWF replacement. Symptoms range from nosebleeds to bleeding from the gums and easy bruising. Bleeding from the stomach and intestines can occur but is less common.
Veyvondi should not be used in the treatment of hemophilia A. It is the first and only recombinant von Willebrand Factor treatment in the European Union for VWD that specifically addresses the primary deficiency or dysfunction of the condition while also allowing the body to restore and maintain adequate Factor VIII (FVIII) plasma levels. Veyvondi is indicated when desmopressin treatment alone is ineffective or not indicated.
“The approval in Europe for Veyvondi marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease,” said Andreas Busch, head of research and development and chief scientific officer, Shire. “We are excited to take the next steps in ensuring that Veyvondi is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement.”
The Marketing Authorization is based on outcomes from three clinical trials of a total 80 patients with VWD exposed to Veyvondi.
With this approval, Shire is now authorized to market VEYVONDI in the 28 Member States of the European Union, as well as in Iceland, Lichtenstein, and Norway.
September 13, 2018
Photo: Andreas Busch, head of research and development and chief scientific officer for Shire