FDA grants Cephalon oncology compound orphan drug designation for AML
The US Food and Drug Administration (FDA) has granted orphan drug designation for lestaurtinib (CEP-701) for the treatment of acute myeloid leukemia (AML).
The US Food and Drug Administration (FDA) has granted orphan drug designation for lestaurtinib (CEP-701) for the treatment of acute myeloid leukemia (AML).
The designation will provide a seven-year period of marketing exclusivity for this indication from the date of final FDA marketing approval of the compound.
Orphan drug designation is given only to certain products intended to treat a rare disease or condition affecting fewer than 200,000 US citizens annually. An estimated 12,000 people in the US were diagnosed with AML in 2005, according to the American Cancer Society. Approximately 25-30% of these patients have an FLT-3 genetic mutation that is associated with a poorer prognosis for relapse and survival.
Lestaurtinib was discovered by international biopharmaceutical company Cephalon, headquartered in Frazer, Pennsylvania. It is a potent inhibitor of several tyrosine kinases including FLT-3 and TrkA and 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 the genetic alteration known as a FLT-3 activating mutation.