FDA Grants Iclusig® Expanded Approval for CML

The United States Food and Drug Administration (FDA) has granted Iclusig® (ponatinib) approval to treat all phases of chronic myeloid leukemia (CML) in adults, as well as Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ALL) who are not eligible for treatment with another tyrosine kinase inhibitor. The approval indication also includes adults with T3151-positive CML and T3151-positive Ph+ ALL.

Both CML and ALL are types of leukemia, which is a type of cancer that starts in blood cells. Ponatinib is referred to as a tyrosine kinase inhibitor (TKI). It has been formulated to block the effects of both BCR-ABL and T3151 pathways in cancer cells, which are implicated in the progression of some leukemias.

Ponatinib was previously approved under the accelerated program of the FDA—a program established so that patients with serious medical conditions can receive earlier access to drugs that appear promising. Under accelerated approval, the FDA requires updated data from clinical trials evaluating the drug prior to granting a full approval.

The full approval of ponatinib was granted by the FDA based on results from 48 months of follow-up from a clinical trial referred to as the PACE clinical trial. This trial included 449 patients with the types of leukemia for which ponatinib is now approved. Patients had received extensive prior therapies and had either stopped responding to treatment, or could not tolerate other available treatment options. The updated data from the PACE trial includes results from 270 patients with chronic-phase CML.

  • 55% of patients achieved a significant reduction in cancer cells detected in the bone marrow (cytogenetic response).
  • 39% of patients achieved a significant reduction in cancer cells detected in the bone marrow, when utilizing sensitive lab tests to detect the genetic abnormalities of their cancer cells.
  • The median duration of both of these types of anti-cancer responses has not yet been reached.
  • Approximately one-third of patients experienced arterial-occlusive events (AOE), which is the narrowing or blockage of one or more arteries.

Patients with all phases of CML, or those with a T3151 mutation, and patients with PH+, or those with a T3151 mutation, now have an additional effective treatment option approved by the FDA.

Patients with these types of leukemias may wish to discuss their personal risks and benefits, as well as eligibility for treatment with ponatinib.

Reference: ARIAD Pharmaceuticals. News Release. ARIAD announces FDA full approval and label update for Iclusig® (ponatinib) based on long-term efficacy and safety data from Phase 2 PACE clinical trial. Available at: http://investor.ariad.com/phoenix.zhtml?c=118422&p=irol-newsArticle&ID=2226080. Accessed December 2, 2016.

 

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