South Korea’s Hanmi Pharmaceutical said Wednesday that its new drug candidate for the treatment of acute myeloid leukemia has been designated as an orphan drug by the US Food and Drug Administration.

Hanmi’s drug candidate in development, HM43239, is a small molecule FMS-like tyrosine kinase 3 inhibitor for the treatment of patients with acute myeloid leukemia. It’s a next-generation drug that has overcome resistance to FLT3 inhibitors currently used to treat the disease, the firm said.


The FDA grants orphan status to drugs and biologics intended for the treatment of rare diseases or life-threatening illnesses that could therefore have limited profitability potential. Benefits include tax breaks, approval fee reductions and a guarantee of seven-year market exclusivity.

With the orphan drug designation, Hanmi is expected to reap benefits in its efforts to further develop and commercialize HM43239 in the US market.

Currently, the US FDA has granted approval to initiate phase 1 clinical trials for HM43238. The first-phase trials are slated to soon start at the MD Anderson Cancer Centers in Houston, Texas, Hanmi said.

This is the third time that a Hanmi-developed drug candidate has obtained orphan drug designation from the FDA. Hanmi’s hyperinsulinemia drug candidate Glucagon Analog (HM15136) and oral cancer drug Oraxol were given orphan drug status in February and April, respectively.

“Hanmi Pharmaceutical’s drug pipeline is steadily growing as diverse new drug candidates, including HM43239, are being treated as rare pharmaceuticals. We will continue to devote ourselves to commercializing these drugs to help patients suffering from diseases,” Hanmi Pharmaceutical CEO Kwon Se-chang said in a statement.

By Sohn Ji-young (jys@heraldcorp.com)