How it is administered

Olutasidenib is taken by mouth as a capsule. Each capsule contains 150 mg of olutasidenib. The usual dose is 150 mg twice daily, taken about the same time each day. It should be swallowed whole and not broken, opened, or chewed. Take olutasidenib on an empty stomach, at least 1 hour before or 2 hours after a meal.

How it works

Olutasidenib is a targeted therapy that works by blocking a mutated enzyme called isocitrate dehydrogenase-1 (IDH1). In some patients with acute myeloid leukemia (AML), mutations in the IDH1 gene cause the enzyme to produce an abnormal substance called 2-hydroxyglutarate (2-HG). This substance can prevent normal blood cell development and contribute to the growth of leukemia cells.

By inhibiting the mutated IDH1 enzyme, olutasidenib lowers the levels of 2-HG in the body. This helps the leukemia cells mature and die, allowing normal blood cells to develop. Olutasidenib is specifically designed for patients whose AML cells have a susceptible IDH1 mutation, and it does not affect the normal (wild-type) IDH1 enzyme or mutated IDH2 enzymes.

Who Should take it

Olutasidenib is indicated for adults with relapsed or refractory acute myeloid leukemia (AML) who have a susceptible IDH1 mutation. This means it is used when AML has come back after previous treatment or has not responded to other treatments, and testing has confirmed the presence of a specific IDH1 mutation in the leukemia cells.

It is important that patients are tested for IDH1 mutations before starting olutasidenib, as the medication is only effective in those with this genetic change. Your healthcare provider will use an FDA-approved test to determine if olutasidenib is appropriate for you.

Who should not take it

There are no absolute contraindications listed for olutasidenib, but it should not be used in patients who are allergic to any of its ingredients.

Caution should be used in patients with severe liver or kidney impairment, as the effects in these populations are not fully known. Women who are pregnant or breastfeeding should not take olutasidenib, as it may cause harm to a developing baby or nursing infant. Always discuss your full medical history with your healthcare provider before starting olutasidenib.

Commonly used with

Olutasidenib is primarily used alone for the treatment of relapsed or refractory AML with IDH1 mutations. In some cases, it may be used alongside supportive care medications such as anti-nausea drugs, antibiotics, or medications to manage blood counts.

It is not indicated for use in combination with other chemotherapy agents for AML, although your healthcare team may use other supportive treatments as needed.

Commonly tested with

Olutasidenib has been studied as a single agent in clinical trials for relapsed or refractory AML with IDH1 mutations. In some studies, it has been tested in combination with azacitidine, but this combination is not currently an approved use.

Routine monitoring with blood tests is required to check for side effects and response to treatment. Your healthcare provider will also test for the IDH1 mutation before starting therapy.