Ponatinib is used in selected hematologic malignancies involving BCR::ABL1, particularly CML and Philadelphia chromosome-positive ALL. The exact eligibility criteria differ by jurisdiction.
Chronic myeloid leukemia
Current U.S. labeling includes:
- Chronic-phase CML with resistance or intolerance to at least two prior kinase inhibitors.
- Accelerated-phase or blast-phase CML for which no other kinase inhibitors are indicated.
- T315I-positive CML in chronic, accelerated or blast phase.
The U.S. label specifically states that ICLUSIG is not indicated or recommended for newly diagnosed chronic-phase CML.
Philadelphia chromosome-positive ALL
U.S. labeling includes ponatinib:
- In combination with chemotherapy for newly diagnosed Ph+ ALL under accelerated approval.
- As monotherapy for Ph+ ALL when no other kinase inhibitors are indicated or for T315I-positive Ph+ ALL.
European authorization
The EMA currently describes Iclusig for adults with CML at different disease stages under specified resistance, intolerance or mutation criteria and for adults with Ph+ ALL under specified circumstances. It also includes monotherapy for children aged six years and older with chronic-phase CML meeting specified criteria.
Approved versus investigational use
Clinical studies involving ponatinib in other diseases do not automatically establish regulatory approval. This website should not present investigational use as an approved treatment.
Treatment selection
The choice of ponatinib depends on leukemia subtype, previous treatment, mutation status, response, comorbidities, cardiovascular risk and the applicable label.