EU regulators reviewing Shire’s bleeding disorder drug

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European regulators have validated the marketing authorisation application for Shire’s experimental von Willebrand Disease therapy Veyvondi.

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.

“With its clinical profile and physiology, Veyvondi is a first-of-its-kind treatment for VWD,” said Alice Dietrich, head of global medical affairs at the firm.

“We’re deeply committed to advancing standards of care and outcomes for VWD patients worldwide, and today’s submission marks an important milestone in our efforts to address needs of patients living with VWD and make VEYVONDI available to more patients who could benefit from its use.”

The treatment was approved by the US Food and Drug Administration as Vonvendi in 2015.

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