Cephalon, Inc. announced that the US Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for lestaurtinib (CEP-701) for the treatment of acute myeloid leukaemia (AML). 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 United States were diagnosed with AML in 2005, according to the American Cancer Society. Approximately 25-30 per cent of these patients have a FLT-3 genetic mutation that is associated with a poorer prognosis for relapse and survival.
Discovered by Cephalon scientists, lestaurtinib is a potent inhibitor of several tyrosine kinases including FLT-3 and TrkA. This orally active, investigational compound is in a phase 2/3 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, the Cephalon release stated.
The Cephalon Oncology portfolio includes three lead compounds targeted for patients with haematologic cancers: Trisenox (arsenic trioxide) injection, an intravenous targeted therapy approved for refractory or relapsed acute promyelocytic leukaemia; Treanda (bendamustine HCl), an investigational multi-functional hybrid cytotoxic in a phase 3 study for relapsed indolent non-Hodgkin's lymphoma; and lestaurtinib.