RIDGEFIELD, Conn. — Boehringer Ingelheim Pharmaceuticals has announced that the Food and Drug Administration and European Commission have granted Orphan Drug Designation to volasertib for acute myeloid leukemia. Volasertib currently is being evaluated in a Phase III clinical trial for the treatment of patients ages 65 years or older with previously untreated AML who are ineligible for intensive remission induction therapy.
Volasertib has not been approved by the 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 primarily is a disease of later adulthood; the average age of an AML patient is 65 years to 70 years. The recommended standard of care currently is intensive chemotherapy, but many patients, due to age and comorbidities, cannot tolerate this therapeutic approach. For them, options are limited, and their prognosis typically is poor. Volasertib currently is being investigated in this specific patient population.
"The FDA and EC Orphan Drug Designations mark an important milestone in our global development program for volasertib," said Sabine Luik, SVP medicine and regulatory affairs, Boehringer Ingelheim Pharmaceuticals. "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."