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

Posted: 17 April 2014 | | No comments yet

Boehringer Ingelheim announced that the US Food and Drug Administration and the European Commission have granted volasertib* ‘orphan drug designation’ for the treatment of patients with acute myeloid leukaemia…

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Boehringer Ingelheim announced today 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 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.1,3 AML is primarily a disease of later adulthood; the average age of an AML patient is 65-70 years.4,5 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.6

Volasertib* inhibits enzymes called Polo-like kinase (Plk).7 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.7,8

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.10

*Volasertib is an investigational compound and is not yet approved. Its safety and efficacy have not yet been fully established.

**The EMA defines a rare disease as one affecting no more than 5 people per 10,000 within the EU. View the full definition here.. Within the US, the FDA considers orphan drug designation status for treatments of diseases affecting fewer than 200,000 people in the U.S., or that affect more than 200,000 people but sales are not expected to recover the costs of developing and marketing the treatment. View the full definition here.


  1. Jemal A, et al. Cancer Statistics, 2009. CA Cancer J Clin. 2009;59:225-249
  2. Ferrara F. Treatment of Unfit Patients With Acute Myeloid Leukemia: A Still Open Clinical Challenge. Clin Lymphoma Myeloma Leuk. 2011;11(1):10-6
  3. National Cancer Institute- Adult Acute Myeloid Leukemia Treatment (PDQ®). Available at: (accessed: February 2014)
  4. Estey EH. Acute myeloid leukemia: 2012 update on diagnosis, risk stratification, and management. Am J Hematol 2012; 87:89-99.
  5. Roboz, Gail J. Novel Approaches to the Treatment of Acute Myeloid Leukemia, Heamatology 2011; 43-50.
  6. Juliusson G, et al. Age and acute myeloid leukemia real world date on decision to treat and outcomes from the Swedish Acute Leukemia Registry. Blood. 2009;113(18):4179-87
  7. Rudolph D, et al. BI 6727, a Polo-like kinase inhibitor with improved pharmacokinetic profile and broad antitumor activity. Clin Cancer Res. 2009;15(9):3094-102.
  8. Schöffski P. Polo-Like Kinase (PLK) Inhibitors in Preclinical and Early Clinical Development in Oncology. Oncologist. 2009;14(6):559-570
  9. Maertens J, et al. Phase I/II Study of Volasertib (BI 6727), an Intravenous Polo-Like Kinase (Plk) Inhibitor, in Patients with Acute Myeloid Leukemia (AML): Results From the Randomized Phase II Part for Volasertib in Combination with Low-Dose Cytarabine (LDAC) Versus LDAC Monotherapy in Patients with Previously Untreated AML Ineligible for Intensive Treatment. Abstract at the 54th Annual Meeting of the American Society of Hematology (ASH) 2012, Atlanta, USA.
  10. Regulation (EC) No 141/2000 of the European Parliament and of the Council.

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