The US Food and Drug Administration (FDA) granted orphan drug designation to gilteritinib for the treatment of acute myeloid leukemia (AML).1
Gilteritinib is an inhibitor of FLT3, a mutated gene commonly found in AML, and AXL, which plays a role in activating FLT3. Gilteritinib has shown inhibitory effects against FLT3 internal tandem duplication (ITD) and tyrosine kinase domain (TKD), 2 frequently occurring FLT3 mutations found in patients with AML.
The FDA based its approval on data from a number of currently underway phase 3 trials, including the ADMIRAL trial (ClinicalTrials.gov Identifier: NCT02421939) for relapsed/refractory FLT3-positive AML.
Data from a previous phase 1/2 trial showed that patients with FLT3+ AML treated with gilteritinib reported a 53% overall response rate (ORR) regardless of prior therapy, and patients who were treated with tyrosine kinase inhibitors prior to the study saw an ORR of 48%. The median overall survival was around 31 weeks.2
Nearly 21,000 patients were diagnosed with AML in 2016, and over 50% of cases resulted in death. Orphan drug designation is granted to therapies intended to prevent, diagnose, or treat conditions that affect fewer than 200,000 people in the US.
- U.S. FDA grants orphan-drug designation to Astellas for development of FLT3 inhibitor gilteritinib in acute myeloid leukemia [news release]. Tokyo, Japan: PRNewswire; July 20, 2017. http://www.prnewswire.com/news-releases/us-fda-grants-orphan-drug-designation-to-astellas-for-development-of-flt3-inhibitor-gilteritinib-in-acute-myeloid-leukemia-300491268.html. Accessed July 20, 2017.
- Astellas announces updated data from the phase ½ chrysalis trial for gilteritinib (ASP2215) in patients with relapsed or refractory acute myeloid leukemia [news release]. Northbrook, IL: PRNewswire; December 6, 2015. http://www.prnewswire.com/news-releases/astellas-announces-updated-data-from-the-phase-12-chrysalis-trial-for-gilteritinib-asp2215-in-patients-with-relapsed-or-refractory-acute-myeloid-leukemia-300188551.html. Accessed July 20, 2017.