FDA grants Breakthrough Therapy designation to Boehringer Ingelheim's volasertib

Boehringer Ingelheim Pharmaceuticals, Inc. today announced the FDA has granted Breakthrough Therapy designation to volasertib, an investigational inhibitor of polo-like kinase (Plk), being evaluated for the treatment of patients aged 65 or older with previously untreated acute myeloid leukemia (AML), ineligible for intensive remission induction therapy.

"This FDA Breakthrough Therapy designation provides Boehringer Ingelheim the opportunity to engage in an ongoing dialogue with the FDA  to help expedite the development of volasertib as a potential treatment option for these patients with AML," said Sabine Luik, M.D., senior vice president, Medicine & Regulatory Affairs, U.S. Regional Medical Director, Boehringer Ingelheim Pharmaceuticals, Inc. "Volasertib is one of many investigational compounds in Boehringer Ingelheim's growing oncology pipeline and is an example of our commitment to exploring treatment approaches with the goal of improving patient outcomes."

The Breakthrough Therapy designation process was established by the FDA in July 2012 and is intended to facilitate and expedite the development and review of drugs for serious or life-threatening conditions if  preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. 

AML is an aggressive cancer of the bone marrow and blood. It accounts for 25 percent of all adult leukemias in the Western world and has one of the lowest survival rates of all leukemias. In 2013, it is estimated there will be 14,590 new cases of AML diagnosed in the U.S. and 10,370 deaths from the disease.

AML is primarily a disease of later adulthood; the average age of an AML patient is 65 years. A common treatment approach for AML is intensive chemotherapy to induce disease remission, followed by consolidation/maintenance chemotherapy.  However, many patients over 65 years of age – whose prognosis is typically poor – are ineligible for this approach, which involves large doses of chemotherapy over short periods of time, and therefore have even fewer treatment options. Volasertib is currently being investigated in this specific patient population.

Results from a Phase II study, in newly diagnosed patients with AML considered ineligible for intensive remission induction therapy, demonstrated higher rates of objective response and an improvement in event-free survival in patients receiving volasertib in combination with low-dose cytarabine (LDAC) compared to patients receiving LDAC alone. The results were presented at the 54th American Society of Hematology (ASH) annual in December 2012.

These results led to the initiation in January 2013 of a Phase III trial, POLO-AML-2 (Clinical Trial Identifier: NCT01721876). This trial is designed to assess the efficacy and safety of volasertib in combination with LDAC, compared to placebo in combination with LDAC, in patients aged 65 or older with previously untreated AML, ineligible for intensive remission induction therapy. The trial is currently enrolling eligible patients.