FDA and EC grant Orphan Drug Designation to Boehringer’s volasertib for acute myeloid leukemia

Boehringer Ingelheim Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (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 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 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 program for volasertib," said Sabine Luik, M.D., 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."

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. To qualify for FDA's Orphan Drug Designation, the drug must, among other requirements, address a disease that affects fewer than 200,000 total people in the U.S. The designation qualifies the sponsor for various development incentives, including tax credits for qualified clinical testing and exemption from user fees under certain circumstances. The Orphan Drug Designation does not change the standard regulatory requirements and process for obtaining market approval. The European Medicines Agency (EMA) defines a rare disease as one affecting no more than five people per 10,000 in the EU.

Following the Breakthrough Therapy Designation granted to volasertib by the FDA in 2013, Boehringer Ingelheim is continuing to engage in dialogue with the 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.