General AML|FLT3

The FDA grants gilteritinib Orphan Drug Designation for the treatment of AML

On 20th July 2017, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to gilteritinib, a Fms Like Tyrosine Kinase 3 (FLT3) inhibitor, for the treatment of patients with Acute Myeloid Leukemia (AML).1

Gilteritinib is a potent, oral FLT3/AXL inhibitor, which binds to and inhibits both the wild-type and mutated forms of FLT3. Recently published results from a phase I/II dose escalation study revealed that gilteritinib monotherapy was well tolerated and generated frequent, prolonged, clinically important responses in FLT3 mutated patients with Relapsed/Refractor (R/R) AML.2

Gilteritinib is currently being investigated in multiple studies including the phase III ADMIRAL study (NCT02421939) which is assessing oral gilteritinib 120 mg/day in R/R AML patients with FLT3 mutations after first-line therapy. The AGP spoke to Alexander E. Perl from the Abramson Comprehensive Cancer Center, University of Pennsylvania, Pennsylvania about the design and plan of the phase 3 ADMIRAL study.

  1. PR Newswire: U.S. FDA Grants Orphan-Drug Designation to Astellas for Development of FLT3 Inhibitor Gilteritinib in Acute Myeloid Leukemia. 2017 July 20. [Accessed 2017 July 20].
  2. Perl A.E. et al. Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1–2 study. Lancet Oncol. 2017 Jun 20. DOI: 10.1016/S1470-2045(17)30416-3. [Epub ahead of print].