Enasidenib for Relapsed or Refractory Acute Myeloid Leukaemia (AML) with an Isocitrate Dehydrogenase 2 (IDH2) Mutation

Acute myeloid leukaemia (AML) is a type of cancer that causes the bone marrow to produce lots of immature white blood cells. It is most common in people aged 60 years and over. Symptoms of AML may include weakness, fatigue, shortness of breath, recurrent infections, prolonged bleeding, loss in appetite and unintended weight loss. Most patients with AML are treated with standard
chemotherapy. AML that is non‐responsive to treatment is called refractory while that which returns after response to initial treatment is called relapsed. Relapsed and refractory AML (rr‐AML) are associated with a poor prognosis. Mutations in a gene called isocitrate dehydrogenase 2 (IDH2) is often present in some patients with AML. There are currently no approved targeted therapies specifically for rr‐AML with an IDH2 mutation.
Enasidenib is a first‐in‐class drug specifically being developed for patients with rr‐AML with an IDH2 mutation. It is a selective inhibitor of mutant‐IDH2 enzymes and acts by blocking the production of chemicals that cause the abnormal (cancerous) white blood cells to grow. Enasidenib is given through the oral route once a day. As there are currently limited treatment options for patients with rr‐AML that have an IDH2 mutation, enasidenib, if licensed, has the potential to offer a new treatment option for this area of high unmet medical need.