How it is administered

Enasidenib is taken by mouth as a tablet. It is available in 50 mg and 100 mg strengths. The usual dose is 100 mg once daily, with or without food. Tablets should be swallowed whole with water and not chewed, split, or crushed. If you miss a dose or vomit after taking a dose, take it as soon as possible on the same day, then return to your normal schedule the next day. Do not take two doses to make up for a missed dose.

How it works

Enasidenib is a targeted therapy that works by inhibiting a specific enzyme called isocitrate dehydrogenase 2 (IDH2). In some blood cancers, such as acute myeloid leukemia (AML), mutations in the IDH2 gene cause the enzyme to function abnormally. This leads to the production of an oncometabolite called 2-hydroxyglutarate (2-HG), which blocks normal cell development and contributes to the growth of cancerous cells.

By blocking the mutant IDH2 enzyme, enasidenib reduces the levels of 2-HG in the body. This helps restore normal cell differentiation, allowing immature blood cells to mature properly. As a result, enasidenib can help decrease the number of cancerous cells and increase the number of healthy blood cells. This mechanism is especially important for patients whose leukemia has returned or did not respond to previous treatments and who have tested positive for an IDH2 mutation.

Who Should take it

Enasidenib is approved for adults with relapsed or refractory acute myeloid leukemia (AML) who have an IDH2 mutation, as detected by an FDA-approved test. This means it is intended for patients whose AML has come back after previous treatment or did not improve with other therapies, and who have a specific genetic change in their cancer cells.

While enasidenib is not specifically approved for myelodysplastic syndromes (MDS), it may be considered in rare cases if MDS progresses to AML with an IDH2 mutation. Your healthcare provider will determine if enasidenib is appropriate for you based on your genetic test results and treatment history.

Who should not take it

There are no absolute contraindications listed for enasidenib, but it should be used with caution in certain situations. Women who are pregnant should not take enasidenib, as it can cause harm to an unborn baby. Effective contraception is recommended for both women and men with partners who could become pregnant during treatment and for at least 2 months after the last dose.

Patients should also inform their doctor about all medications and supplements they are taking, as enasidenib can interact with other drugs. If you have a history of severe allergic reactions to any of the ingredients in enasidenib, you should not take this medication. Safety and effectiveness in children have not been established.

Commonly used with

Enasidenib is typically used as a single agent (monotherapy) for patients with relapsed or refractory AML with an IDH2 mutation. In some cases, it may be used alongside supportive treatments such as hydroxyurea to control high white blood cell counts or corticosteroids to manage differentiation syndrome.

It is important to discuss all other medications you are taking with your healthcare provider, as enasidenib can interact with several other drugs, including certain antifungals, antibiotics, and medications that affect liver enzymes.

Commonly tested with

Enasidenib has been studied primarily as a single agent in clinical trials for relapsed or refractory AML with an IDH2 mutation. In these studies, it was not commonly combined with other anti-leukemia drugs, but supportive medications such as hydroxyurea and corticosteroids were sometimes used to manage side effects or complications.

Ongoing research may explore combinations with other targeted therapies or chemotherapy, but for now, its main use is as a single agent in the specific patient population described.