Shire Says EMA Validates Veyvondi MAA For Treatment Of Von Willebrand Disease

(RTTNews) - Shire plc (SHPG, SHP.L) said that the European Medicines Agency or EMA validated the Marketing Authorization Application or MAA for VEYVONDI to prevent and treat bleeding episodes and peri-operative bleeding in adults (age 18 and older) diagnosed with von Willebrand Disease or VWD, the most common inherited bleeding disorder.

Currently available in the U.S. as VONVENDI [von Willebrand factor (Recombinant)], VEYVONDI is the first and only recombinant von Willebrand factor (rVWF) treatment for adults living with VWD.

Patients with VWD have a deficiency or dysfunction of VWF, a blood protein required for proper clotting. Because of this, the blood does not clot properly, which may result in heavy menstrual periods, easy bruising, or frequent nose bleeds.

VEYVONDI is an innovative 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.

VEYVONDI was studied in patients, 18 to 64 years of age, in a multi-center, open label, non-randomized study assessing safety, efficacy and pharmacokinetics, with and without rFVIII. It was also studied in patients, 18 years and older, in a prospective, uncontrolled, international, multi-center, open label, non-randomized study assessing control of hemostasis before, during or after surgical procedures, with or without rFVIII.

The most common adverse reaction observed was generalized pruritus (itching). The EMA filing is based on data from these two Phase 3 clinical trials shared publicly in December 2015 and December 2016, respectively.