Takeda has recently announced that the U.S. Food and Drug Administration approved the supplemental New Drug Application (sNDA) for ICLUSIG® (ponatinib). This approval expands the usage of ICLUSIG to treat adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) when used alongside chemotherapy. The approval, granted under accelerated approval, is based on achieving minimal residual disease (MRD)-negative complete remission (CR) at the end of the induction phase. Continued approval for this indication will depend on the verification and description of clinical benefits in confirmatory trials. This application received Priority Review and was evaluated under the Real-Time Oncology Review (RTOR) program, aimed at expediting the delivery of cancer medications by allowing partial review before the submission of a complete application.

Dr. Awny Farajallah, Takeda's chief medical officer for oncology, expressed enthusiasm about this milestone, highlighting the significance of providing a targeted treatment option for U.S. adult patients with newly diagnosed Ph+ ALL. The FDA's recognition of ICLUSIG's potential to address a critical gap in patient care is seen as promising for individuals with this rare and aggressive cancer.

The approval was based on data from the PhALLCON study, the first Phase 3, global, registrational clinical trial comparing ICLUSIG to imatinib in adults with newly diagnosed Ph+ ALL. Patients treated with ICLUSIG demonstrated superior outcomes, achieving more than double the rate of MRD-negative CR compared to those treated with imatinib at the end of the induction phase. Safety profiles of ICLUSIG and imatinib were comparable, with no new safety concerns identified during the trial.

Dr. Elias Jabbour, lead investigator of the PhALLCON trial from The University of Texas MD Anderson Cancer Center, emphasized the urgent need for potent tyrosine kinase inhibitors (TKIs) to improve outcomes in Ph+ ALL patients. He believes that ponatinib could address these needs and potentially impact long-term outcomes.

ICLUSIG, a kinase inhibitor, is now approved in the U.S. for newly diagnosed Ph+ ALL in combination with chemotherapy, under accelerated approval based on achieving MRD-negative CR at the end of induction. Its use as monotherapy is also approved in various Ph+ ALL and chronic myeloid leukemia (CML) settings. The drug's efficacy against resistant mutations, including the T315I mutation, makes it a valuable option for patients who have failed other TKIs.

The PhALLCON trial, a Phase 3 study, evaluated the efficacy and safety of ICLUSIG compared to imatinib in combination with reduced-intensity chemotherapy as a frontline therapy for adult patients with newly diagnosed Ph+ ALL.

Ph+ ALL is a rare form of leukemia characterized by the presence of the Philadelphia chromosome. It affects approximately a quarter of adult ALL patients in the U.S. and is associated with poor outcomes. ICLUSIG targets the abnormal tyrosine kinase BCR::ABL1, expressed in CML and Ph+ ALL, making it a targeted therapy designed to inhibit the activity of this kinase and its mutations.

ICLUSIG received full FDA approval in November 2016 and has since been a critical option for patients with various forms of Ph+ ALL and CML.

 

Source: takeda.com

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