How it is administered

Ivosidenib is taken by mouth as a film-coated tablet. The recommended dose is 500 mg orally once daily, with or without food, but you should avoid taking it with a high-fat meal. Swallow the tablets whole; do not split, crush, or chew them. If a dose is missed or vomited, do not take an extra dose—just take the next scheduled dose as usual.

How it works

Ivosidenib is a targeted therapy that works by inhibiting a specific mutated enzyme called isocitrate dehydrogenase 1 (IDH1). In certain blood cancers like myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), mutations in the IDH1 gene can cause abnormal cells to produce a substance called 2-hydroxyglutarate (2-HG). This substance interferes with normal cell development and leads to the growth of cancerous cells.

By blocking the activity of the mutant IDH1 enzyme, ivosidenib reduces the levels of 2-HG. This allows abnormal blood cells to mature and function more normally, which can help control the disease and improve blood counts. Ivosidenib is only effective in patients whose cancer cells have the susceptible IDH1 mutation, so testing is required before starting treatment.

Who Should take it

Ivosidenib is indicated for adults with relapsed or refractory myelodysplastic syndromes (MDS) who have a susceptible IDH1 mutation, as detected by an FDA-approved test. It is also used in certain patients with acute myeloid leukemia (AML) and in some cases of cholangiocarcinoma (a type of bile duct cancer) with the same mutation.

For MDS, it is specifically for patients whose disease has returned after or did not respond to previous treatments. Your healthcare provider will test your blood or bone marrow to confirm the presence of the IDH1 mutation before prescribing ivosidenib.

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 components. Caution is advised in patients with a history of long QT syndrome, uncontrolled heart disease, or those taking other medications that can prolong the QT interval, as ivosidenib can increase the risk of serious heart rhythm problems.

Women who are pregnant or breastfeeding should not take ivosidenib, as it may cause harm to the unborn baby or nursing infant. Discuss all medications and health conditions with your doctor before starting ivosidenib.

Commonly used with

In the treatment of newly diagnosed AML, ivosidenib is commonly used in combination with azacitidine, another medication that helps control abnormal blood cells. For MDS and relapsed/refractory AML, it is typically used as a single agent.

Your doctor may also prescribe supportive treatments such as antibiotics, antifungals, or medications to manage side effects, depending on your individual needs.

Commonly tested with

Ivosidenib has been tested in combination with azacitidine for newly diagnosed AML. For MDS and relapsed/refractory AML, it has been studied as a single agent. It is also tested alongside standard supportive care medications.

Before starting ivosidenib, your healthcare team will test for the IDH1 mutation using an FDA-approved diagnostic test. Regular blood tests and heart monitoring (ECG) are also performed during treatment to monitor for side effects and effectiveness.