Common brand names

• Iclusig

How it is administered

Ponatinib is taken by mouth as a tablet. It is available in strengths of 10 mg, 15 mg, 30 mg, and 45 mg. The tablets should be swallowed whole and can be taken with or without food. Do not crush, break, cut, or chew the tablets. If a dose is missed, take the next dose at the regularly scheduled time the next day. Do not take two doses at the same time to make up for a missed dose.

How it works

Ponatinib is a type of medicine called a kinase inhibitor. It works by blocking the activity of certain proteins called kinases, specifically the BCR::ABL protein, which is produced by abnormal genes in some leukemia cells. This protein helps cancer cells grow and multiply. Ponatinib is particularly effective against cancer cells with a mutation called T315I, which makes them resistant to many other treatments.

By blocking these proteins, ponatinib helps to stop the growth and spread of leukemia cells. It also inhibits other kinases that may play a role in cancer cell survival. This makes ponatinib useful for people whose chronic myeloid leukemia (CML) has not responded to other treatments, or who have a specific mutation that makes their cancer harder to treat.

Who should take it

Ponatinib is used to treat adults with chronic myeloid leukemia (CML) who have not responded to at least two other kinase inhibitors, or who have a specific mutation called T315I. It is also used for people with more advanced phases of CML (accelerated or blast phase) when no other kinase inhibitors are suitable, and for those with T315I-positive CML in any phase.

Ponatinib may also be used in certain cases of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), but for CML, it is specifically for those who have resistance or intolerance to other treatments. It is not recommended for people with newly diagnosed chronic phase CML.

Who should not take it

Ponatinib does not have any absolute contraindications listed, but it should not be used in people who are allergic to ponatinib or any of its ingredients.

Caution is needed for people with a history of heart disease, high blood pressure, blood clots, pancreatitis, liver problems, or those who are pregnant or breastfeeding. Ponatinib can cause serious side effects, so your doctor will carefully consider the risks and benefits before starting treatment, especially if you have risk factors for blood clots or heart problems.

Commonly used with

For chronic myeloid leukemia, ponatinib is usually used alone (monotherapy), especially in patients who have not responded to other treatments. In some cases, for other conditions like Ph+ ALL, it may be used in combination with chemotherapy.

Your doctor will decide if ponatinib should be used alone or with other medications based on your specific situation and previous treatments.

Commonly tested with

Ponatinib has been tested in clinical studies both as a single agent and in combination with chemotherapy, especially for Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). For chronic myeloid leukemia, it is mainly studied as monotherapy in patients who have failed other kinase inhibitors or have the T315I mutation.

It is not typically combined with other targeted therapies for CML, but your healthcare team may monitor you closely and adjust your treatment plan as needed.