Gilteritinib gains orphan-drug status to combat AML

Gilteritinib works by inhibiting certain receptors involved in cancer cell growth. | File photo

Because acute myeloid leukemia (AML) impacts relatively few individuals nationwide, gilteritinib — an anti-cancer medication developed by Tokyo-based Astellas Pharma Inc. — recently attained orphan-drug designation from the U.S. Food and Drug Administration.

Orphan-drug designation is assigned to those medications addressing rare diseases — quantified as affecting fewer than 200,000 individuals in the U.S. AML is a cancer affecting blood and bone marrow, mostly in older adults.

Gilteritinib works by inhibiting certain receptors involved in cancer cell growth and has been shown to hinder FLT3 and AXL mutations observed in approximately one-third of AML victims. Astellas continues investigations of the drug in clinical trials.

"Fewer than 10,000 Americans will be diagnosed with FLT3 mutation-positive AML this year and while that may be a small percentage of the overall population, it is an important group of patients who are deserving of potential new treatments,” Dr. Steven Benner, Astellas senior vice president and global therapeutic area head for the company’s oncology development, said. “We are grateful to the FDA for acknowledging the unique needs of rare diseases and for providing a path forward for gilteritinib in supporting these patients.”

Astellas officials confirmed that gilteritinib ranks as the company’s first fast-track venture, meaning of highest priority and expedited for research and development with extra resources.

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