FDA grants gilteritinib Fast Track Designation for the treatment of R/R AML

On 10th October 2017, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to gilteritinib, a Fms Like Tyrosine Kinase 3 (FLT3) inhibitor, for the treatment of adult patients with FLT3 mutation positive (FLT3+) Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML).1 This comes after the recent Orphan Drug Designation for gilteritinib granted by the FDA in July 2017.

Gilteritinib is a potent, oral FLT3/AXL inhibitor, which binds to and inhibits both the wild-type and mutated forms of FLT3. Data from a phase I/II dose escalation study (NCT02421939), which was published in Lancet Oncology (as reported by the AGP) revealed that gilteritinib monotherapy was well tolerated and generated frequent, prolonged, clinically important responses in FLT3 mutated patients with R/R AML.2 Additionally in an interview with the AGP,  Alexander E. Perl from the Abramson Comprehensive Cancer Center, University of Pennsylvania said that gilteritinib showed “limited efficacy in patients that did not have FLT3 mutations suggesting that this agent is highly selective in its activity against the FLT3 inhibition”.

Presently, gilteritinib is being explored 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.

  1. PR Newswire: U.S. FDA Grants Fast Track Designation to Astellas for Development of Gilteritinib in Relapsed or Refractory Acute Myeloid Leukemia. 2017 Oct 10. http://www.prnewswire.com/news-releases/us-fda-grants-fast-track-designation-to-astellas-for-development-of-gilteritinib-in-relapsed-or-refractory-acute-myeloid-leukemia-300534294.html. [Accessed 2017 Oct 11].
  2. Perl A.E. 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; 18: 1061–-75