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FDA grants orphan drug designation to gilteritinib for acute myeloid leukemia
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The FDA granted orphan drug designation to gilteritinib for the treatment of acute myeloid leukemia, according to the agent’s manufacturer.
Gilteritinib (ASP2215, Astellas), a receptor tyrosine kinase inhibitor of FLT3 and AXL, appeared active against FLT3 internal tandem duplication and tyrosine kinase domain, two common types of FLT3 mutations observed among patients with AML.
Phase 3 trials designed to evaluate the safety and efficacy of gilteritinib in different AML patient populations are underway. These include the registrational ADMIRAL trial, which includes patients with relapsed/refractory FLT3-positive AML.
“Fewer than 10,000 Americans will be diagnosed with FLT3 mutation-positive AML this year and, [although] that may be a small percentage of the overall population, it is an important group of patients who are deserving of potential new treatments,” Steven Benner, MD, senior vice president and global therapeutic area head of oncology development at Astellas, said in a company-issued press release. “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.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.