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].
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