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18 Apr 2014

Volasertib?, Boehringer Ingelheim'?s Investigat?ional Oncology Compound, is Granted Orphan Drug Designatio?n for Acute Myeloid Leukemia in the US and EU

Boehringer Ingelheim Pharmaceuticals, Inc. has announced that FDA and European Commission (EC) have granted Orphan Drug Designation to volasertib for acute myeloid leukemia (AML).


Volasertib is currently being evaluated in a Phase III clinical trial for the treatment of patients aged 65 or older, with previously untreated AML, who are ineligible for intensive remission induction therapy. Volasertib has not been approved by FDA or EC regulatory authorities; its safety and efficacy have not been established.


AML is an aggressive cancer of the bone marrow and blood. It accounts for approximately one-third of all adult leukemias in the Western world and has one of the lowest survival rates of all leukemias. 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 comorbidities cannot tolerate this therapeutic approach. For them, options are limited and their prognosis is typically poor. Volasertib is currently being investigated in this specific patient population.


"The FDA and EC Orphan Drug Designations mark an important milestone in our global development programme for volasertib," said Sabine Luik, MD, senior vice president, Medicine & Regulatory Affairs,  Boehringer Ingelheim Pharmaceuticals, Inc. "Boehringer Ingelheim is deeply committed to supporting the community of patients living with acute myeloid leukemia, with a current research focus on these older patients who have very limited treatment options."


Following the Breakthrough Therapy Designation granted to volasertib by FDA in 2013, Boehringer Ingelheim is continuing to engage in dialogue with FDA to help expedite the development of volasertib as a potential treatment option. Publication of the Phase I/II trial data that was used in support of the Breakthrough Therapy Designation is expected later this year.

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