Volasertib* receives FDA Breakthrough Therapy Designation for treatment of patients with acute myeloid leukaemia

Boehringer Ingelheim plans to work closely with the US Food and Drug Administration (FDA) to potentially provide a new treatment option to improve survival related outcomes for patients with acute myeloid leukaemia (AML)

INGELHEIM, Germany - Tuesday, September 17th 2013 [ME NewsWire] (BUSINESS WIRE)--For non-U.S. media only
Boehringer Ingelheim is pleased to announce that the FDA has granted a Breakthrough Therapy Designation to volasertib*, a selective and potent polo-like kinase (Plk) inhibitor, for the treatment of patients with AML, a type of blood cancer.
“Volasertib’s* innovative mode of action offers a new approach and may potentially provide a new therapy option for AML patients who have a high unmet medical need,” said Professor Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim. “We are looking forward to exploring opportunities with the FDA to expedite the final stage of the development of volasertib* with the aim of making this treatment available for patients.”
The Breakthrough Therapy designation pathway was launched by the US Food and Drug Administration (FDA) in 2012, and is intended for any drug that “treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.”1
A Phase II study in patients with previously untreated AML ineligible for intensive therapy compared volasertib* in combination with the established therapy of low-dose cytarabine (LDAC) versus LDAC alone.2 The primary endpoint for the Phase II study was objective response. Objective responses were observed in 31 percent of patients (13 of 42 patients) treated with the combination of volasertib* plus LDAC compared to 13.3 percent of the patients (6 of 45 patients) treated with LDAC alone (p = 0.0523).
Secondary endpoints included event-free survival (EFS), overall survival (OS) and safety. A trend for OS benefit (8.0 months for the volasertib* combination compared to 5.2 months for LDAC alone, p = 0.0996) was observed. EFS was measured from the date of randomisation to the date of disease progression (treatment failure), relapse or death from any cause, whichever occurred first. In patients treated with the combination of volasertib* plus LDAC, the median EFS was 5.6 months compared to 2.3 months in patients treated with LDAC alone (hazard ratio: 0.56; 95% CI: 0.34, .93; p=0.0237).
These encouraging results led to the initiation of the Phase III study, POLO-AML-2, investigating volasertib* in combination with LDAC, in patients aged 65 years and above with previously untreated AML who are ineligible for intensive remission induction therapy.
“The most common treatment approach for AML is intensive remission induction therapy; however, many older patients are ineligible for this approach which involves high doses of chemotherapy which these patients are often unable to tolerate,” said Professor Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim, “These patients are in particular need of new treatments and we hope that volasertib* may be able to fill this gap by providing a tolerable option that improves survival related outcomes.”
Please click on the link below for ‘Notes to Editors’ and ‘References’:
http://www.boehringer-ingelheim.com/news/news_releases/press_releases/2013/17_september_2013oncology.html
*Volasertib is an investigational compound and is not yet approved. Its safety and efficacy have not yet been fully established.
References
1 FDA Frequently Asked Questions: Breakthrough Therapies. http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/ucm341027.htm [Last accessed: June 2013]
2 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. Oral Presentation at ASH Annual Meeting and Exposition 2012
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