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, or chew the tablets.

How it works

Ponatinib is a type of medication called a kinase inhibitor. It works by blocking the action of specific proteins called kinases, which are involved in the growth and survival of cancer cells. In particular, ponatinib targets the BCR-ABL protein, including forms of this protein that have mutations (such as T315I) that make them resistant to other treatments.

By inhibiting these kinases, ponatinib helps to stop the growth and spread of cancer cells in certain blood cancers. It also affects other kinases involved in blood vessel formation and cell signaling, which may contribute to its effectiveness in treating blood cancers like chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

Who Should take it

Ponatinib is used to treat adults with specific types of blood cancers:

• Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL): It is used in combination with chemotherapy for newly diagnosed patients, and as a single agent for patients who have no other kinase inhibitor options or have the T315I mutation.
• Chronic myeloid leukemia (CML): It is indicated for patients with chronic phase CML who are resistant or intolerant to at least two prior kinase inhibitors, for those with accelerated or blast phase CML for whom no other kinase inhibitors are indicated, and for those with the T315I mutation.

Ponatinib is not recommended for patients with newly diagnosed chronic phase CML.

Who should not take it

There are no absolute contraindications listed for ponatinib. However, it should be used with caution in patients who have a history of serious heart or blood vessel problems, uncontrolled high blood pressure, severe liver problems, or a history of pancreatitis. Women who are pregnant or breastfeeding should not use ponatinib, as it can harm the unborn baby or nursing infant.

Patients with active, uncontrolled cardiovascular disease, recent heart attack, or stroke, and those with severe kidney or liver impairment should discuss risks and benefits with their doctor before starting ponatinib.

Commonly used with

For newly diagnosed Ph+ ALL, ponatinib is commonly used in combination with chemotherapy agents such as vincristine, dexamethasone, methotrexate, cytarabine, and prednisone as part of a treatment regimen.

In other indications, ponatinib may be used alone (monotherapy) when other kinase inhibitors are not suitable.

Commonly tested with

Ponatinib has been tested in clinical trials alongside chemotherapy agents for Ph+ ALL, including vincristine, dexamethasone, methotrexate, cytarabine, and prednisone. It has also been compared to other kinase inhibitors like imatinib in clinical studies.

In CML, ponatinib is generally tested as a single agent in patients who have not responded to other treatments.