Ivosidenib for acute myeloid leukaemia with IDH1 mutation

Ivosidenib is an oral treatment in clinical development for people with acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation. AML is a type of cancer that causes the bone marrow to produce excess immature white blood cells. 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. AML in general, and particularly relapsed or refractory disease, is associated with a poor prognosis. In blood cancers such as AML, certain genetic mutations such IDH1 can occur.
Ivosidenib belongs to a new class of therapies that works by inhibiting the mutated IDH1 enzyme, which in turn reduces the level of d-2-hydroxyglutarate (2-HG), an oncometabolite which impairs myeloid differentiation, increases proliferation of myeloblasts, and blocks cellular differentiation. There are currently no approved treatment options in the EU/UK for those who have relapsed or refractory AML with an IDH1 mutation. If licensed, ivosidenib could be an effective precision medicine for this patient group.