CHMP recommends approval of Veyvondi in von Willebrand disease.- Baxalta.

The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Veyvondi (vonicog alfa), from Baxalta, intended for the treatment of von Willebrand disease. Veyvondi was designated as an orphan medicinal product on 26 November 2010. Veyvondi will be available as a powder and solvent for solution for injection (650 IU and 1300 IU). The active substance of Veyvondi is vonicog alfa, a recombinant human von Willebrand factor which behaves in the same way as endogenous von Willebrand factor (ATC code: B02BD10).

The benefits with Veyvondi are its ability to re-establish platelet adhesion to the endothelium at the site of blood vessel damage and to correct associated factor VIII deficiency. The most common side effects are dizziness, vertigo, dysgeusia, tremor, tachycardia, deep venous thrombosis, hypertension, hot flush, vomiting, nausea, generalised pruritus, chest discomfort, infusion site paraesthesia, electrocardiogram T wave inversion and increased heart rate.

The full indication is: "Veyvondi is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the: �Treatment of haemorrhage and surgical bleeding �Prevention of surgical bleeding. Veyvondi should not be used in the treatment of Haemophilia A."

Comment: VWD is a hereditary bleeding disorder that is caused by deficiency or dysfunction of VWF, a plasma protein that mediates the initial adhesion of platelets at sites of vascular injury and also carries and protects factor VIII from premature proteolysis. Because of this, the blood does not clot properly, resulting in heavy menstrual periods, easy bruising, or frequent nose bleeds. It is estimated that one in 100 people, approximately 3 million people in the United States, suffer from VWD.