This morning, Astellas Pharmaceuticals announced that its compound gilteritinib was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for use in acute myeloid leukemia (AML) patients.
Gilteritinib is a receptor tyrosine kinase inhibitor of FLT3 and AXL, which are heavily responsible for the expansion of cancer cells. In May, data were presented at the 2017 American Society of Clinical Oncology (ASCO 2017) showing that the administration of gilteritinib was correlated with clinical response and improved overall survival in patients with FLT3 mutation-positive leukemia.
AML is a cancer in which the bone marrow produces abnormal myeloblasts, or unipotent stem cells. Symptoms most frequently include fatigue, recurrent infections and easy bruising. It affects both the blood and bone marrow, and is most frequently diagnosed in older adults.
Per the American Cancer Society, there were an estimated 21,000 new cases of AML diagnosed in the United States, and about half resulted in death.
“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,” said Steven Benner, M.D., senior vice president and global therapeutic area head, oncology development of Astellas in a press release.
Gilteritinib has shown inhibitory activity against FLT3 internal tandem duplication, in addition to tyrosine kinase domain, 2 FLT3 mutations present in approximately 1/3 of AML patients.
Currently, the agent is being studied by Astellas in an array of AML patient populations via planned and already underway Phase 3 trials, including the ADMIRAL trial in relapsed/refractory FLT3+ AML.
At ASCO 2017, Rare Disease Report sat down with Dr Daniel DeAngelo, MD, PhD of the Dana Farber Cancer Institute to discuss AML, and the unmet needs throughout the patient community.
Source > http://www.raredr.com/news/orphan-drug-designation-gilteritinib-aml