In December 2015, a new drug was approved in the USA for patients suffering from a rare form of von Willebrand disease. Due to an inherited defect, von Willebrand factor in these patients is altered to such a degree that massive and dangerous bleeding can occur. In healthy patients, von Willebrand factor ensures that blood platelets in the circulation are immediately recruited to repair blood vessels as soon as they are injured. The new drug, VONVENDI was developed by Baxalta (previously Baxter, now Shire) and is the first, and so far only, recombinant agent for the treatment of von Willebrand disease. In clinical studies it demonstrated a 100% treatment success rate. VONVENDI is based on a substance patent belonging to the MDC. Several years ago scientists there examined active agents that could be used for the treatment of bleeding disorders, including von Willebrand factor. At that time, a substance patent was applied for and subsequently licensed to Baxter by the MDC with the help of Ascenion in 2010.
(Annual Review 2015)
