Cephalon has announced that the FDA has granted orphan-drug designation for its leukemia treatment candidate lestaurtinib.
The compound inhibits several tyrosine kinases, including FLT-3 and TrkA, believed to be involved in some cancers. Cephalon is developing lestaurtinib as an oral treatment for acute myeloid leukemia (AML), a disease that afflicted 12,000 people in the U.S. last year.
Lestaurtinib is currently in a Phase II/III trial in AML patients who have relapsed from standard induction chemotherapy and who also have a FLT-3 genetic mutation. Approximately 25 percent to 30 percent of AML patients have this genetic mutation, which is associated with an increased risk for relapse and a poorer prognosis for survival.