The US Food and Drug Administration (FDA) and the European Commission have granted orphan drug designation for Boehringer Ingelheim's volasertib for the treatment of patients with acute myeloid leukaemia (AML).

AML is an aggressive cancer of the bone marrow and blood. It accounts for approximately one third of all adult leukaemias in the Western world and has one of the lowest survival rates of all leukaemias. AML is primarily a disease of later adulthood; the average age of an AML patient is 65-70 years. 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). 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). 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.

Professor Klaus Dugi, chief medical officer at Boehringer Ingelheim commented, "We are pleased that both the FDA and European Commission have decided to grant orphan drug designation to volasertib. This coupled with the FDA Breakthrough Therapy Designation awarded to the compound last year, recognises the potential of volasertib as a possible new treatment for patients with acute myeloid leukaemia (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. In parallel with the on-going phase III trials, we will work closely with both agencies and hope patients will benefit from our medicine as soon as possible."

The phase I/II clinical trial of volasertib combined with chemotherapy improved survival times for elderly patients with AML9. Publication of the full results of the phase I/II clinical trial is expected later this year.

In both the US and the EU, 'orphan drug designation’ is awarded to medicines intended to treat rare conditions that have limited treatment options and where currently no authorised treatment and/or diagnosis method exists. It indicates that regulatory support and incentives will be offered to the company to help the development and authorisation process.

Volasertib,a selective and potent Plk inhibitor, is currently being evaluated in clinical trials for AML and is one of two late-stage compounds that Boehringer Ingelheim is currently evaluating in clinical trials for cancer. It is currently in phase III development. The phase III study, POLO-AML-2, is investigating volasertib in combination with low-dose cytarabine (LDAC, a form of chemotherapy), in patients aged 65 years and above with previously untreated AML who are ineligible for intensive remission induction therapy.