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Boehringer´s Volasertib for Acute Myeloid Leukaemia is Granted ‘Orphan Drug Designation’ in the EU and the US

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  • An orphan drug is a medicine that treats a rare disease

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

  • 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 options

April 17, 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. 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 AML. 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.

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