FDA approves Takeda’s Iclusig for Philadelphia chromosome-positive ALL

Twelve years after the FDA initially granted limited approval to Takeda’s Iclusig (ponatinib) for treating specific patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL), the FDA has now expanded its approval for broader use. Takeda announced this new endorsement, allowing the drug to be combined with chemotherapy for newly diagnosed patients with the disorder, marking an accelerated approval process that is subject to further confirmation of clinical benefits in subsequent trials. This milestone makes Iclusig the first and sole targeted therapy available for these patients in the United States, according to Takeda.
The original approval for Iclusig more than a decade ago was for its standalone use and was restricted to patients who were unable to use other tyrosine kinase inhibitors (TKIs) or those with T315I-positive Ph+ALL. Awny Farajallah, Takeda’s chief medical officer, expressed excitement about this label expansion, highlighting its potential to address a significant gap in care for patients with this rare and aggressive form of cancer.

The label expansion follows a head-to-head trial involving 245 patients who received either Iclusig or Novartis’ first-generation TKI Gleevec (imatinib) alongside reduced-intensity chemotherapy. During a 12-week induction period, the group receiving Iclusig showed promising results, with 34.4% of patients achieving undetectable disease levels compared to 16.7% in the Gleevec group. Furthermore, compared to 26% in the Gleevec group, only 7% of patients in the Iclusig group stopped their medication due to ineffectiveness.
The PhALLCON trial’s principal investigator, Elias Jabbour, underlined the long-standing requirement for a strong TKI that can inhibit the emergence of mutations and generate significant responses in frontline therapy. He proposed that ponatinib, the active component of Iclusig, might address these important variables and enhance patients’ long-term results.
The Philadelphia chromosome gene is an uncommon marker for Ph+ALL, a rare disease that affects about 25% of adult ALL patients in the United States. According to the American Cancer Society, there will be 6,550 new cases of Ph+ALL in the United States in 2024 alone, and 1,330 people will pass away from this extremely aggressive form of cancer. In addition to its approval for Ph+ALL, Iclusig is also indicated for patients with chronic-phase myeloid leukemia who have shown resistance or intolerance to at least two prior kinase inhibitors. Originally developed by Ariad Pharmaceuticals, Takeda acquired the Massachusetts-based cancer specialist for $5.2 billion in 2017.

In a recent report, Takeda disclosed that Iclusig generated sales of 41,460 million Japanese yen ($275 million) in the previous three quarters.