Volasertib, Boehringer Ingelheim´s investigational treatment of acute myeloid leukaemia, is granted ‘orphan drug designation’ in the EU and the US

On 17 April 2014 Boehringer Ingelheim reported that the US Food and Drug Administration (FDA) and the European Commission have granted volasertib orphan drug designation for the treatment of patients with acute myeloid leukemia (AML) (Press release Boehringer Ingelheim, APR 16, 2014, View Source [SID:1234500431]).
Volasertib inhibits enzymes called Polo-like kinase (Plk). Plk1 is the best characterised kinase of the Plk family. Inhibition of Plk1 by volasertib results in blocking the cell cycle, ultimately leading to cell death (apoptosis).7 By inhibiting Plk1 activity, the extremely high cell division that is characteristic of AML should be blocked, which may result in stopping the tumour cell growth and even could lead to a reduction in actively dividing tumour cells. Ultimately, this could allow patients to live longer.