Hanmi Pharmaceutical said Wednesday that its HM43239, a drug candidate in treating acute myeloid leukemia (AML), has won orphan drug status from the U.S. Food and Drug Administration.
The HM43239 is a candidate to overcome drug resistance of FLT3 inhibitors while inhibiting AML-induced FLT3 (FMS-like tyrosine kinase 3) mutations. Hanmi confirmed the superior efficacy of HM43239 in various animal experiments using FLT3-mutated AML cell lines, and plans to enter phase 1 clinical phase in the U.S. MD Anderson Cancer Center.
FDA’s Orphan Drug Designation is a system that helps smooth the development and approval of therapeutic drugs for rare or life-threatening diseases. ODD drugs receive tax holidays, exemption of license application fees, and seven-year monopoly after obtaining authorization.
With the ODD status for HM43239, Hanmi has a total of three ODD drugs, including HM15136, a congenital hyperinsulinemia treatment, and Oraxol, an oral anticancer drug for angiosarcoma.
“Hanmi's pipeline is expanding steadily, as HMD3232 and other Hanmi Pharm's new drugs are receiving ODD status,” Hanmi Pharmaceutical’s President-CEO Kwon Se-chang said. “Our company will speed up development for patients suffering from the disease.”