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Boehringer's AML Drug Gains Orphan Status

April 17, 2014

Volasertib being evaluated in patients with limited treatment options

Boehringer Ingelheim’s volasertib for acute myeloid leukemia (AML) has been granted Orphan Drug Designation by the FDA and European Commission. Volasertib is being evaluated in a Phase III trial for the treatment of patients aged 65 or older, with previously untreated AML, who are ineligible for intensive remission induction therapy.
 
Volasertib is an investigational compound that inhibits Plk1 enzymes, which regulates cell division and can result in prolonged cell cycle arrest, ultimately leading to cell death. AML is an aggressive cancer of the bone marrow and blood and has one of the lowest survival rates of all leukemias.
 
In both the U.S. and EU, Orphan Drug Designation is a status given to investigational compounds intended to treat a rare disease or condition that has limited treatment options.
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