New Drug Approved by European Commission

The European Commission has granted marketing authorization for caplacizumab for the treatment of adults experiencing an episode of aTTP on August 31, 2018.

From: Caplacizumab, the active substance in Cablivi, is a nanobody (a small antibody) which has been designed to attach to von Willebrand factor in a way that stops it acting on platelets. This reduces platelets sticking together and forming clots in blood vessels and, as a result, platelet levels in the blood rise because they are no longer taken up to form clots.

The European Medicines Agency decided that Cablivi’s benefits are greater than its risks and it can be authorised for use in the EU. The Agency considered that, in patients with aTTP, Cablivi combined with plasma exchange and immunosuppression can reduce the time it takes for platelet counts to return to the normal range, which is associated with shorter duration of plasma exchange treatment and shorter stay in intensive care facility.The most important side effect of treatment is bleeding but it is considered manageable. The company is expected to provide results of a study on Cablivi’s safety and effectiveness over a longer period.

From: drug has been accepted for priority review in the United States and the Food and Drug Administration is expected to make a decision by Feb. 6, 2019.