Takeda Receives FDA Approval for ICLUSIG as a First-Line Treatment in Ph+ ALL

Takeda Pharmaceutical Company Limited (TSE: 4502; NYSE: TAK) is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to a Supplemental New Drug Application (sNDA) for ICLUSIG (ponatinib), in combination with chemotherapy, for the treatment of adult patients newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). This landmark approval introduces ICLUSIG as the first and only first-line targeted therapy for Ph+ ALL in combination with chemotherapy in the United States, based on achieving minimal residual disease (MRD)-negative complete response (CR).

The FDA's decision marks the first time a therapy for Ph+ ALL has been approved based on MRD-negative CR, a significant advancement in the treatment paradigm for this aggressive cancer. This accelerated approval was facilitated by priority review and the Real-Time Oncology Review (RTOR) program, reflecting the drug’s potential to fill a significant treatment gap and improve patient outcomes.

PhALLCON Study Drives Approval

The approval of ICLUSIG is supported by robust data from the PhALLCON Phase 3 trial, a pivotal study that demonstrated ICLUSIG's superiority over imatinib in achieving higher rates of MRD-negative CR when used in combination with low-intensity chemotherapy. The study highlighted ICLUSIG's potential to offer a more effective treatment option for patients newly diagnosed with Ph+ ALL, achieving a two-fold increase in MRD-negative CR rates at the end of the induction phase. Furthermore, ICLUSIG's safety profile was found to be comparable to that of imatinib.

A Milestone for Patient Care

Awny Farajallah, M.D., Chief Medical Officer of Oncology at Takeda, expressed enthusiasm about the label expansion, emphasizing its significance in providing a targeted therapy option for patients facing this challenging diagnosis. "This is a tremendous milestone in our ongoing efforts to bring innovative treatments to patients with Ph+ ALL. ICLUSIG’s approval for first-line use, in combination with chemotherapy, underscores its potential to significantly impact patient care and improve long-term outcomes," said Dr. Farajallah.

Elias Jabbour, MD, Principal Investigator of the PhALLCON trial and a faculty member at the University of Texas MD Anderson Cancer Center, echoed the sentiment, highlighting the urgent need for effective treatments in Ph+ ALL. "ICLUSIG's ability to inhibit the development of mutations and elicit a deep response offers hope for patients with this very aggressive form of cancer," stated Dr. Jabbour