On 1st December 2017, the US Food and Drug Administration (FDA) granted crenolanib, a tyrosine kinase inhibitor, Fast Track designation for the treatment of patients with Fms Like Tyrosine Kinase 3 (FLT3) mutation-positive Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML).1 FLT3 mutations with Internal Tandem Duplications (ITD) occurs in approximately 30% of AML patients. These mutations often lead to poor prognosis and disease relapse in AML patients.2
Crenolanib is a selective and potent inhibitor for Platelet-Derived Growth Factor Receptor (PDGFR) α and β but it also has a high affinity for FLT3. Crenolanib binds to and inhibits both wild-type and mutated forms of FLT3, which can lead to the inhibition of FLT3-related signal transduction pathways.2
At present, crenolanib is being explored in multiple trials for newly diagnosed (NCT02283177) and relapsed/refractory (NCT02298166, NCT02400281, NCT01657682) AML patients with or without FLT3 mutations.