Indications of Ponatinib

Chronic Myeloid Leukemia (CML) or Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ ALL)

1. Indicated for the treatment of adult patients with T315I mutation-positive chronic myeloid leukemia in chronic phase, accelerated phase or blast phase. It is also indicated for adult patients with chronic myeloid leukemia in chronic phase who have developed resistance or intolerance to at least two prior kinase inhibitors, as well as those with chronic myeloid leukemia in accelerated phase or blast phase for whom no other suitable kinase inhibitors are available.

2. Not recommended for the treatment of newly diagnosed chronic myeloid leukemia in chronic phase.

3. Can be used as a monotherapy for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia who have no other applicable kinase inhibitors, or for those with T315I mutation-positive Philadelphia chromosome-positive acute lymphoblastic leukemia.

4. Can also be used in combination with chemotherapy for the treatment of newly diagnosed patients with Philadelphia chromosome-positive acute lymphoblastic leukemia. This indication was granted accelerated approval based on the results of minimal residual disease (MRD)-negative complete remission at the end of induction therapy. The continued approval of this indication is contingent upon the verification of clinical benefit in confirmatory clinical trials.

5. This product has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the aforementioned indications.

FDA,2022.02