Regulators on both sides of the Atlantic have awarded orphan drug status to Boehringer Ingelheim's leukaemia drug volasertib.
Both the European Medicines Agency and US Food and Drug Administration have awarded orphan designation to the drug as a treatment for patients with acute myeloid leukaemia (AML) who are not eligible for intensive treatment.
AML is an aggressive cancer of the bone marrow and blood which accounts for around one third of all adult leukaemias in the Western world and has one of the lowest survival rates.
Volasertib inhibits Polo-like kinase (Plk) enzymes to block the cell cycle and induce cell death, thus interrupting the extremely high cell division that is characteristic of AML. This, says Boehringer, could even lead to a reduction in actively dividing tumour cells and allow patients to live longer.
According to the firm, a Phase I/II clinical trial, full data from which will be available later this year, has shown that the combing the drug with chemotherapy improved survival for elderly patients with AML.
"Due to the targeted way in which volasertib works, we hope it will offer a new alternative for those patients who are currently left with limited options," noted Professor Klaus Dugi, the German drugmaker's Chief Medical Officer.
The drug also has Breathrough Therapy status in the US.