Lestaurtinib granted orphan drug status

Published: 1-May-2006
Regulatory

The US FDA has granted orphan drug designation to lestaurtinib (CEP-701) for the treatment of acute myeloid leukemia (AML), giving it seven years of marketing exclusivity from final marketing approval.


The US FDA has granted orphan drug designation to lestaurtinib (CEP-701) for the treatment of acute myeloid leukemia (AML), giving it seven years of marketing exclusivity from final marketing approval.

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 have a FLT-3 genetic mutation associated with a poorer prognosis for relapse and survival.

Lestaurtinib, discovered by US biopharma company Cephalon, is a potent inhibitor of several tyrosine kinases, including FLT-3 and TrkA, and 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 in those whose disease presents with a FLT-3 activating mutation.

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