EMA grants Orphan Drug Designation to gilteritinib for the treatment of AML

On 23rd January 2018,  the European Medicines Agency’s (EMA)  Committee for Orphan Medicinal Products (COMP) 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 This comes after gilteritinib was granted Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA) in July and October 2017 respectively.

Gilteritinib is a potent, oral FLT3/AXL inhibitor, which binds to and inhibits both the wild-type and mutated forms of FLT3. Findings 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, data from an on-going phase I dose-escalation study  (NCT02236013), presented at the 59th American Society of Hematology (ASH) Annual Meeting by Keith Pratz, MD, from the John Hopkins University, demonstrated that gilteritinib in combination with intensive chemotherapy was “well tolerated” with a maximum tolerated dose at ≥ 120 mg/day in patients with newly diagnosed AML. A higher response for this combination regimen was seen in FLT3mut+ AML patients.3 In an interview with the AGP, Keith Pratz highlighted that the responses observed in patients treated in this phase I dose escalation trial has been “good so far”.

At present, 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 CenterUniversity of Pennsylvania, about the design and plan of the phase III ADMIRAL study.

  1. Astellas: Astellas Receives Orphan Designation from the European Commission for Gilteritinib for the Treatment of Acute Myeloid Leukaemia. 2018 Jan 23. https://www.astellas.com/en/news/10211 [Accessed 2018 Jan 23].
  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].
  3. Pratz K. et al. Preliminary Results from a Phase 1 Study of Gilteritinib in Combination with Induction and Consolidation Chemotherapy in Subjects with Newly Diagnosed Acute Myeloid Leukemia (AML). Oral Abstract #722: 59th ASH Annual Meeting and Exposition, Atlanta, GA.
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