OSAKA, Japan & CAMBRIDGE, Mass. - Takeda Pharmaceutical Company (NYSE:TAK) Limited (TSE:4502/NYSE:TAK) announced today that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib) in combination with chemotherapy for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).
This indication has been granted accelerated approval based on the achievement of minimal residual disease (MRD)-negative complete remission (CR) rates at the end of induction therapy. The approval is contingent upon verification of clinical benefit in confirmatory trials.
The approval of ICLUSIG for this indication is significant as it offers a targeted treatment option for adult patients with Ph+ ALL, a rare and aggressive cancer. The FDA's decision was based on results from the Phase 3 PhALLCON trial, where ICLUSIG demonstrated superiority in MRD-negative CR rates compared to imatinib, another treatment option.
The trial's primary endpoint was met, showing more than a two-fold improvement in MRD-negative CR rates at the end of induction (cycle 3) for patients treated with ICLUSIG. The safety profile of ICLUSIG was comparable to imatinib, with no new safety signals identified.
The PhALLCON trial is a global, randomized, open-label multicenter study that evaluated the efficacy and safety of ICLUSIG versus imatinib, each in combination with reduced-intensity chemotherapy.
A total of 245 patients were enrolled in the trial, with a median age of 54 for the ICLUSIG group and 52 for the imatinib group. The primary endpoint was the rate of MRD-negative CR at the end of induction therapy, with event-free survival as a key secondary endpoint.
Ph+ ALL is characterized by the presence of an abnormal gene known as the Philadelphia chromosome, affecting approximately 25% of adult ALL patients in the U.S. ICLUSIG is a kinase inhibitor designed to inhibit the activity of BCR::ABL1, an abnormal tyrosine kinase expressed in CML and Ph+ ALL, including the T315I mutation, which is associated with resistance to other approved tyrosine kinase inhibitors (TKIs).
The approval of ICLUSIG is based on a press release statement from Takeda and marks an important development for patients with this challenging form of leukemia.
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