Korean Pharma company, Hanmi has won orphan drug status this week from the US Food and Drug Administration (FDA) for HM43239, a drug for the treatment of acute myeloid leukaemia (AML).
The endorsement of FDA’s Orphan Drug Designation (ODD) — invented to smooth the development of therapies for life-threatening diseases that are rare with a small patient group — will dramatically speed up approval for Hanmi’s drug.
The candidate HM43239 is designed to overcome drug-resistant FLT3 inhibitors while inhibiting AML-induced FLT3 (FMS-like tyrosine kinase 3) mutations.
Hanmi announced the drug’s success in a number of animal experiments using FLT3-mutated AML cell lines and plans to enter phase 1 clinical phase in the US MD Anderson Cancer Center.
Hanmi’s pipeline is growing steadily. In addition to HM43239, the company has a total of three ODD drugs, including HM15136, a congenital hyperinsulinemia treatment, and Oraxol, an oral anticancer drug for angiosarcoma.