The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to volasertib for acute myeloid leukemia. 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. The agent has not been approved by the FDA regulatory authorities, and its safety and efficacy have not been established.
The recommended standard of care for AML 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.
Volasertib is an investigational compound that inhibits enzymes called Polo-like kinase (Plk). Inhibition of Plk1, the best characterized kinase of the Plk family, can result in prolonged cell cycle arrest, ultimately leading to apoptosis. Publication of the phase I/II trial data that was used in support of the Breakthrough Therapy Designation is expected later this year. ■