Boehringer Ingelheim receives orphan drug designation for volasertib in US and EU

Volasertib is an investigational compound that inhibits enzymes called Polo-like kinase. Currently, the drug is being assessed in a Phase III clinical trial for the treatment of patients aged 65 or above, with previously untreated AML, who are ineligible for intensive remission induction therapy.

Safety and efficacy of volasertib have not been established and the drug is currently not approved by the FDA and European Commission.

Boehringer Ingelheim Pharmaceuticals medicine and regulatory affairs senior vice president Dr Sabine Luik noted the FDA and EC Orphan Drug Designations mark an important milestone in the company’s 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," Dr Luik added.

In 2013, Boehringer Ingelheim has received Breakthrough Therapy Designation from the FDA for volasertib. Following the Breakthrough Therapy Designation, the company 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 2014.

Currently, volasertib is being evaluated in clinical trials for various solid tumors and hematological cancers.

POLO-AML-2, an ongoing global Phase III trial, has been initiated in January 2013 to evaluate the safety and efficacy of volasertib in combination with low-dose cytarabine (LDAC), compared to placebo in combination with LDAC, in patients aged 65 or above with previously untreated AML, ineligible for intensive remission induction therapy. The Phase III trial is currently enrolling eligible patients.