How it is administered

Ivosidenib is taken by mouth as a film-coated tablet. The usual dose is 500 mg once daily, with or without food, but you should avoid taking it with a high-fat meal. Tablets should be swallowed whole and not split, crushed, or chewed. If you miss a dose and it is more than 12 hours until your next dose, take it as soon as possible. If you vomit a dose, do not take an extra dose—just take your next dose at the scheduled time.

How it works

Ivosidenib is a targeted therapy that works by blocking a specific abnormal enzyme called isocitrate dehydrogenase 1 (IDH1), which is found in some people with acute myeloid leukemia (AML) and other cancers. When IDH1 is mutated, it causes the production of an abnormal substance called 2-hydroxyglutarate (2-HG), which interferes with normal cell development and leads to the growth of cancerous cells.

By inhibiting the mutated IDH1 enzyme, ivosidenib reduces the levels of 2-HG, allowing the leukemia cells to mature and die naturally, rather than continue to grow uncontrollably. This helps to restore normal blood cell production and can lead to remission of the leukemia. Ivosidenib is specifically used in patients whose cancer cells have the IDH1 mutation, which is determined by a special test.

Who Should take it

Ivosidenib is used for adults with acute myeloid leukemia (AML) who have a specific mutation in the IDH1 gene, as detected by an FDA-approved test. It can be used in newly diagnosed AML patients who are 75 years or older, or who have other health conditions that make intensive chemotherapy too risky. It can also be used in adults with AML that has come back (relapsed) or has not responded to previous treatments (refractory).

Additionally, ivosidenib is approved for use in adults with relapsed or refractory myelodysplastic syndromes (MDS) with an IDH1 mutation, and for certain patients with advanced cholangiocarcinoma (bile duct cancer) who have been previously treated and have an IDH1 mutation.

Who should not take it

There are no absolute contraindications listed for ivosidenib, but it should not be used in patients who are allergic to any of its ingredients. Caution is needed in patients with a history of long QT syndrome, uncontrolled heart problems, or those who are taking other medications that can prolong the QT interval, as ivosidenib can increase the risk of dangerous heart rhythm problems.

Women who are pregnant or breastfeeding should not take ivosidenib, as it may cause harm to a developing baby or nursing infant. The safety and effectiveness of ivosidenib in children have not been established.

Commonly used with

In newly diagnosed AML, ivosidenib is often used in combination with azacitidine, especially for patients who cannot tolerate intensive chemotherapy. In other cases, it may be used alone (monotherapy).

It may also be used alongside supportive care medications, such as those to manage infections, nausea, or low blood counts.

Commonly tested with

Ivosidenib has been tested in combination with azacitidine for newly diagnosed AML with an IDH1 mutation. It has also been studied as a single agent in both newly diagnosed and relapsed/refractory AML and MDS.

In clinical trials, it is sometimes tested with other supportive medications, but the main combination in AML is with azacitidine.