Boehringer Ingelheim's volasertib granted orphan drug designation for AML

The US Food and Drug Administration and the European Commission have granted German family-owned drug major Boehringer Ingelheim orphan drug designation for volasertib for the treatment of patients with acute myeloid leukemia (AML).

AML is an aggressive cancer of the bone marrow and blood which accounts for about one third of all adult leukemias in the Western world and has one of the lowest survival rates of all leukemias. The recommended standard of care is currently intensive chemotherapy, but many patients due to age and co-morbidities cannot tolerate this therapeutic approach. For them, options are limited and their prognosis is poor.

Volasertib inhibits enzymes called Polo-like kinase (Plk), the best characterized kinase of the Plk family. This results in blocking the cell cycle, ultimately leading to cell death (apoptosis) and by inhibiting Plk1 activity, the extremely high cell division that is characteristic of AML should be blocked, which may result in stopping the tumor cell growth and even could lead to a reduction in actively dividing tumor cells. Ultimately, this could allow patients to live longer.