Shire gets European thumbs-up for VWD bleeding disorder treatment

Shire has received a marketing authorisation application from the European Medicines Agency for doctors to use its Veyvondi treatment on adult patients with the bleeding disorder von Willebrand Disease.

Veyvondi is the first recombinant von Willebrand factor (rVWF) in Europe, Shire pointed out, and is indicated for the prevention and treatment of bleeding episodes and peri-operative bleeding episodes.

EMA approval follows US approval under the name of Vonvendi in 2015 and comes on the back of two Phase 3 trials published in December 2015 and 2016.

US regulators approved the drug as an on-demand treatment for controlling bleeding episodes in patients with VWD and Shire is seeking to expand the indication for prophylaxis and paediatric populations, with trials supporting the applications due to conclude in 2019 and 2020 respectively.

Patients with VWD have a deficiency or dysfunction of a blood protein known as VWF required for proper clotting, resulting in blood not clotting properly and often leading to heavy menstrual periods, easy bruising or frequent nose bleeds.

Shire's treatment looks to replace VWF "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 [Factor VIII - an essential blood-clotting protein also known as anti-hemophilic factor], which is said "provides physicians with the flexibility to manage VWF levels".

"With its clinical profile and physiology, Veyvondi is a first-of-its-kind treatment for VWD," said Alice Dietrich, Shire's head of global medical affairs, adding that the submission marks "an important milestone" in efforts to address needs of patients living with VWD.