Oscotec said Thursday that its SKI-G-801, an FMS (Feline McDonough Sarcoma)-like tyrosine kinase 3 (FLT3) target acute leukemia treatment, has won orphan drug status from the U.S. Food and Drug Administration.

FDA’s orphan drug designation (ODD) 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.

SKI-G-801 is a next-generation drug candidate that can overcome the drug resistance of existing FLT3 inhibitors by actively inhibiting the FLT3 mutation, a common gene that causes acute myelogenous leukemia.

Currently, five clinical trials are underway in clinical laboratories, including the Rosewell Park Cancer Institute.

About 30 percent of patients with acute myelogenous leukemia have an FLT3 mutation. SKI-G-801 has shown excellent efficacy against various mutants such as FLT3-ITD, FLT3 D839Y, and FLT3 F691L, while the company expects that it will exhibit an excellent anti-cancer effect even in non-reactive patients, who have relapsed after using existing drugs or do not show any efficacy from such medications.

In the clinical trials, 10 to 20 patients who were unresponsive to the existing drug or who had relapsed after the previous drug administration will receive a dose escalation test to confirm the safety and preliminary efficacy. Afterward, the company plans to expand the trial by administering the safe dosage to 20 FLT3 mutation patients.

According to the company, the global market for acute leukemia therapy reached $450 million in 2015, and as it has shown an average annual growth rate of 17.32 percent, the market will reach $1 billion by 2020.

In particular, with the low survival rate of elderly patients with acute myelogenous, the company expects that if the new drug is successful, the survival rate will dramatically increase significantly expanding the market.

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