FDA Grants Accelerated Approval to Ponatinib in Ph+ ALL

The US Food and Drug Administration (FDA) has granted accelerated approval to ponatinib with chemotherapy for adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

Efficacy was evaluated in the phase III PhALLCON study, a randomized, active-controlled, multicenter, open-label trial of 245 adult patients with newly diagnosed Ph+ ALL. Patients were randomized to receive either ponatinib 30 mg orally once daily or imatinib 600 mg orally once daily with chemotherapy. Chemotherapy consisted of three cycles of induction with vincristine and dexamethasone, six cycles of consolidation alternating between methotrexate and cytarabine, and 11 cycles of maintenance with vincristine and prednisone. The ponatinib dose was reduced to 15 mg once daily after completion of the induction phase and achievement of measurable residual disease (MRD)-negative complete remission (CR).

Efficacy was based on the MRD-negative CR rate at the end of induction. The MRD-negative CR rate at the end of induction was 30% in the ponatinib arm and 12% in the imatinib arm. Treatment

“The potential benefit to patients who achieve this early deep response may be important to improve long-term survival outcomes,” said lead author Elias Jabbour, MD, from the University of Texas MD Anderson Cancer Center, in a 2023 press release. “The trial results indicate ponatinib as a potential standard of care for patients newly diagnosed with Ph+ ALL.”

Related: Elias Jabbour, MD, on the Phase III PhALLCON Trial in Ph+ ALL