Takeda Submits sNDA for Iclusig Supported by Five-Year Survival Data Showing 93% of CML Patients Alive at Two Years

Compiled by Keke

Approximately one-third of patients with chronic myeloid leukemia (CML) do not survive beyond five years, partly due to increased resistance to treatment regimens. Takeda seeks to change this status quo.

On Monday, December 7, Takeda presented a set of long-term analysis results from the Phase 2 clinical trial Optic for Iclusig at the 62nd Annual Meeting of the American Society of Hematology (ASH). The data showed that among CML patients who had previously received two or more tyrosine kinase inhibitors and were treated with Iclusig, more than 50% remained alive five years after initiating treatment at a dose of 45 mg/day, with no disease progression. The study also indicated that at the two-year mark, 93% of patients in the same cohort were still alive, and 81% showed no evidence of cancer progression.

These findings stem from a subset analysis of the Phase 2 Optic trial, highlighting an improved benefit-risk profile for Iclusig, a third-generation tyrosine kinase inhibitor (TKI), in patients with chronic-phase chronic myeloid leukemia (CP-CML) who have failed prior second-generation TKI therapy, regardless of mutation status or resistance. The analysis also included an assessment of the patient population following treatment with second-generation TKIs. Although CML is generally manageable, patients who have experienced treatment failure on prior second-generation TKIs, particularly those who have developed resistance, face poorer long-term prognoses. This underscores the persistent gaps in the care of patients with CML.

Takeda hopes that drug regulatory authorities will recognize the strengths of these data. Warren stated, “The company has submitted a supplemental new drug application to the U.S. Food and Drug Administration (FDA) and has been granted priority review. The recently released data will serve as the foundation to support our submission of the approval application.”

Ponatinib is an oral drug developed by Ariad Pharmaceuticals. In 2017, Takeda acquired Ariad for $5.2 billion, thereby obtaining this drug. Certain forms of chronic myeloid leukemia (CML) harbor the T315I mutation and are resistant to current therapies such as imatinib. Ponatinib is a multi-targeted tyrosine kinase inhibitor (TKI) that primarily targets BCR-ABL, an abnormal tyrosine kinase that is a hallmark of CML and Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Ponatinib was designed to effectively combat these types of malignancies and has currently been approved for the treatment of patients with CML and Ph+ ALL.

The drug was first approved for marketing in the United States in December 2012, but its sales were suspended on October 31, 2013, due to the “risk of life-threatening thrombosis and severe vascular stenosis.” On December 20, 2013, the sales suspension was partially lifted, and revised prescribing information for ponatinib was issued, including a new “Boxed Warning” and “Risk Evaluation and Mitigation Strategy (REMS),” to better assess the risks and benefits of using this medication. If Takeda obtains approval for the supplemental application for this drug, it will continue to expand the use of Iclusig in chronic myeloid leukemia (CML).

Reference Source:

1. Wikipedia

2.ASH: Takeda pads case for new leukemia use with 5-year survival data

3.Takeda Oncology Demonstrates Leadership in Hematologic Cancers at 62nd American Society of Hematology (ASH) Annual Meeting