European regulators have approved Shire’s von Willebrand Disease therapy Veyvondi.
The green light allows doctors to prescribe the therapy for the treatment of bleeding events and treatment/prevention of surgical bleeding in adults with the condition, when desmopressin treatment alone is ineffective or not indicated.
VWD is the most common bleeding disorder worldwide, affecting up to one in 100 people and its rarest form one in 1,000,000.
The genetic disorder causes alterations or deficits in von Willebrand factor, resulting in impaired clotting, and can manifest through a variety of bleeding events such as mucosal bleeds or menorrhagia.
Patients often live with the disease for years without a proper diagnosis, and even with a confirmed diagnosis, there are limited treatment options available.
Veyvondi is a VWF replacement therapy produced and formulated without the addition of any exogenous raw materials of human or animal origin, resulting in a product that contains only trace amounts of FVIII. This provides physicians with the flexibility to manage VWF levels, Shire says.
“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 R&D and chief scientific officer at the firm.
“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 treatment was approved by the US Food and Drug Administration under the brand name Vonvendi in 2015.