Regulatory approval

Published by the Food and Drug Administration.

The U.S. Food and Drug Administration granted accelerated approval to ponatinib in combination with chemotherapy for the treatment of adult patients with newly diagnosed philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The product label notes that this indication is approved under accelerated approval based on minimal residual disease (MRD)-negative complete remission (CR) at the end of induction and that continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial(s). This indication is based on PhALLCON (NCT03589326), a randomized, active-controlled, multicenter, open-label trial in which chemotherapy regimens were: vincristine and dexamethasone (induction, cycles 1 to 3); methotrexate and cytarabine (consolidation, cycles 4 to 9, alternating methotrexate and cytarabine); and vincristine and prednisone (maintenance, cycles 10 to 20).

This is written in the approval document as:

ICLUSIG is a kinase inhibitor indicated for the treatment of adult patients with newly diagnosed Ph+ ALL, in combination with chemotherapy. This indication is approved under accelerated approval based on minimal residual disease (MRD)-negative complete remission (CR) at the end of induction. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial(s).

Citation

Takeda Pharmaceuticals America, Inc. Iclusig (ponatinib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/203469s037lbl.pdf. Revised March 2024. Accessed October 30, 2024.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Type	Biomarker(s)	Cancer type	Therapy(ies)
Sensitivity (+)	BCR::ABL1	Acute Lymphoid Leukemia	Cytarabine, Methotrexate, Ponatinib, Prednisone, Vincristine