Target drugs

Target drugs focus on particular component within the leukaemia cell. Target drugs may be added to the induction treatment, when remission has not been achieved. Several targeted drugs that are effective include:

  • Midostaurin (RydaptÒ, Novartis Europharm Limited) specifically blocks the mutation in the FLT3 gene in patients with AML. It is called a FLT3 inhibitor.
  • When given in combination with daunorubicin+cytarabine, midostaurin is approved for the treatment of adult patients with newly‑diagnosed AML with a FLT3 mutation.
  • In combination with high-dose cytarabine, midostaurin is also approved as consolidation treatment.
Gemtuzumab ozogamicin
  • Gemtuzumab ozogamicin (MylotargÒ, Pfizer/Wyeth-Ayerst Laboratories) is a monoclonal antibody linked to calicheamicin, which is a cytotoxic drug. Cytotoxic means toxic to cells.
  • Monoclonal antibodies are laboratory-produced proteins that bind to certain targets on the surface of the leukaemia cells.
  • Gemtuzumab ozogamicin is approved for use with daunorubicin and cytarabine in patients with newly diagnosed AML that shows the CD33 protein
  • Gilteritinib (XospataÒ, Astellas Pharma Europe B.V) is an oral, potent and selective FLT3 inhibitor with efficacy in relapsed or refractory FLT3 AML.
  • It is approved as treatment on its own for the treatment of adult patients who have relapsed or refractory AML with a FLT3 mutation.
  • Glasdegib (DaurismoÒ, Pfizer Limited UK) inhibits a protein in the Hedgehog signalling pathway making the leukaemia cells more sensitive to chemotherapy.
  • The Hedgehog signalling pathway normally transmits information to the body’s cells on how to develop and mature. Mutation or disturbance of this pathway plays a key role limiting the development of leukaemia cells.
  • Glasdegib is used in combination with low-dose cytarabine, for newly‑diagnosed AML patients older than 75 years, or patients who cannot withstand intensive induction chemotherapy.