FDA And EMA Grant Orphan Drug Status To BI's AML Drug Volasertib

Boehringer Ingelheim announced that it has received Orphan Drug Designation for volasertib from both the U.S. Food and Drug Administration (FDA) and the European Commission. Volasertib is intended for the treatment of acute myeloid leukemia and is currently undergoing assessment in a Phase III trial.

Sabine Luik, SVP of Medicine & Regulatory Affairs at Boehringer Ingelheim, said, “The FDA and EC Orphan Drug Designations mark an important milestone in our global development program for volasertib. 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.”

Acute myeloid leukemia is an aggressive form of cancer affecting the bone marrow and the blood. AML has one of the lowest survival rates of all leukemias and typically affects patients in later adulthood. Due to age and comorbidities, patients often cannot tolerate intensive chemotherapy. Volasertib is being assessed as potential treatment for the elderly patient population.

The investigational compound inhibits enzymes called Polo-like kinase (Plk), currently the best characterized kinase of the Plk family. Plk1 regulates cell division. Inhibition of Plk1 can lead to a prolonged cell cycle arrest, ultimately resulting in cell death (apoptosis).

Volasertib is being evaluated in a Phase III clinical trial for the treatment of patients 65 years or older with previously untreated AML and who are ineligible for intensive remission induction therapy.

Orphan Drug Designation is granted to investigational compounds intended to treat rare disease or conditions that have limited treatment options. In the U.S., an orphan drug candidate must address a disease that afflicts fewer than 200,000 people. In the EU, the EMA defines a rare disease as one that afflicts no more than five people per 10,000. An Orphan Drug status qualifies the sponsor company for various development incentives including tax credits and user fee exemptions.

The drug also received Breakthrough Therapy Designation from the FDA in 2013.