• Although a rare disease, acute myeloid leukaemia is one of the most common adult leukaemias

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• EMA & FDA recognise volasertib's promise as a potential new treatment for patients with acute myeloid leukaemia who are not eligible for intensive treatment
• Currently ~40% of AML patients are unlikely to benefit from intensive therapy options2

For media outside of the US only

Ingelheim, Germany, 17 April 2014 – 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

Professor Klaus Dugi, Chief Medical Officer at Boehringer Ingelheim

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

Notes to editors:

About volasertib*

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.11

About Acute Myeloid Leukaemia (AML)
AML is a rare cancer of the bone marrow and blood. It is one of the most common types of acute leukaemia in adults, accounting 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 predominantly occurs in older adults; the average age of newly diagnosed patients is 65- 70 years.4,5 In AML patients the prognosis worsens with increasing age, with a median survival less than a year12 following diagnosis in older patients who are often ineligible for intensive remission induction therapy (the most common treatment approach for younger patients).4,13 This involves high doses of chemotherapy which older patients often are unable to tolerate.

About Boehringer Ingelheim in Oncology
Building on scientific expertise and excellence in the fields of pulmonary and cardiovascular medicine, metabolic disease, neurology, virology and immunology, Boehringer Ingelheim has embarked on a major research programme to develop innovative cancer drugs. Working in close collaboration with the international scientific community and a number of the world’s leading cancer centres, Boehringer Ingelheim’s commitment to oncology is underpinned by using advances in science to develop a range of targeted therapies for various solid tumours and haematological cancers.

The current focus of research includes compounds in three areas: angiogenesis inhibition, signal transduction inhibition and cell-cycle kinase inhibition. Volasertib* is the most advanced pipeline compound for haematological cancers. The pipeline for haematological cancers also includes two new biological entities (NBEs) using an immunotherapeutic approach: BI 836858 is an anti-CD33 antibody targeting AML and BI 836826 is an anti-CD37 antibody targeting chronic lymphocytic leukaemia (CLL) and B-cell non-Hodgkin's lymphoma (B-NHL).

Boehringer Ingelheim’s oncology pipeline is evolving and demonstrates the company’s continued commitment to advance the disease area.

Boehringer Ingelheim
The Boehringer Ingelheim group is one of the world's 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, Boehringer Ingelheim operates globally with 142 affiliates and a total of more than 47,400 employees. The focus of the family-owned company, founded in 1885, is researching, developing, manufacturing and marketing new medications of high therapeutic value for human and veterinary medicine.

Taking social responsibility is an important element of the corporate culture at Boehringer Ingelheim. This includes worldwide involvement in social projects, such as the initiative "Making more Health" and caring for the employees. Respect, equal opportunities and reconciling career and family form the foundation of the mutual cooperation. In everything it does, the company focuses on environmental protection and sustainability.

In 2013, Boehringer Ingelheim achieved net sales of about 14.1 billion euros. R&D expenditure corresponds to 19.5% of its net sales.

*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.

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References
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: http://www.cancer.gov/cancertopics/pdq/treatment/adultAML/healthprofessional/page1/AllPages (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.
11. Information on volasertib phase III study, POLO-AML-2, available at: http://clinicaltrials.gov/ct2/show/NCT01721876?term=POLO-AML-2&rank=1 (Last viewed: 12 Mar 2014)
12. Juliusson G, et al. Acute myeloid leukemia in the real world: why population-based registries are needed. Blood. 2012;119(17):3890-9
13. Fredly H, et al. Combination of the histone deacetylase inhibitor valproic acid with oral hydroxyurea or 6-mercaptopurin can be safe and effective in patients with advanced acute myeloid leukaemia--a report of five cases. Hematology 2010;15:338-343.

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