Cephalon leukemia drug granted orphan status in US

The orphan drug designation will provide a seven-year period of marketing exclusivity for this indication from the date of final FDA marketing approval of the compound.

FDA may designate a medication as an orphan drug if the product is intended to treat a rare disease or condition affecting less than 200,000 Americans annually.

An estimated 12,000 people in the US were diagnosed with acute myeloid leukemia (AML) in 2005, according to the American Cancer Society. Approximately 25-30% of these patients have a FLT-3 genetic mutation that is associated with a poorer prognosis for relapse and survival.

Cephalon’s drug candidate is a potent inhibitor of several tyrosine kinases, including FLT-3 and TrkA. The orally active, investigational compound is in a phase II/III clinical trial. It is a targeted agent against AML in patients at first relapse from standard induction chemotherapy and whose disease presents with a genetic alteration known as a FLT-3 activating mutation.