Thursday, April 17, 2014
FDA Grants AML Drug Volasertib Orphan Drug Status
The U.S. Food and Drug Administration has granted orphan drug designation to volasertib for the treatment of acute myeloid leukemia (AML). The drug—an investigational inhibitor of polo-like kinase (Plk)—is currently being evaluated for safety and efficacy in a Phase III clinical trial for the treatment of patients who are 65 or older with previously untreated AML who are ineligible for intensive remission induction therapy.
The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
About 18,860 new cases of AML are expected to be diagnosed this year, according to estimates from the American Cancer Society. The average age at diagnosis is 66.
Volasertib (Boehringer Ingelheim Pharmaceuticals, Inc.) received the FDA’s Breakthrough Therapy Designation in 2013 based on Phase I and II trial data (OT 10/25/13 issue), which is expected to be published later this year, according to a news release from the drug company.