FDA Approves Iclusig for New Patients with Aggressive Leukemia


The accelerated approval of Iclusig for patients with acute lymphoblastic leukemia and with the Philadelphia chromosome was based on a surrogate endpoint. Iclusig has a list price of $20,831 for 30 tablets.

The FDA has granted an accelerated approval for a supplemental indication for Iclusig (ponatinib). It is now approved to treat adult patients who have been newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL). Iclusig is indicated to be used in combination with chemotherapy.

Philadelphia-positive ALL is a type of fast-growing blood cancer that starts in young white blood cells called lymphoblasts or blast cells. About 25% of adult ALL patients in the United States have the abnormal Philadelphia chromosome. In these patients, an abnormal chromosome is formed when pieces of chromosomes 9 and 22 switch with each other. Patients with Ph+ ALL receiving only chemotherapy have a high rate of relapse.

Elias Jabbour, M.D.

Elias Jabbour, M.D.

“Ph+ ALL is an extremely aggressive cancer, and patients with this disease suffer from poor outcomes,” Elias Jabbour, M.D., The University of Texas MD Anderson Cancer Center and lead investigator said in a press release.

Developed by Takeda, Iclusig is an oral kinase inhibitor that targets BCR-ABL1, an abnormal tyrosine kinase. Iclusig is already approved to treat patients who in are the chronic phase of chronic myelogenous leukemia and the blast phase of Philadelphia chromosome-positive acute lymphoblastic leukemia.

Iclusig has a wholesale acquisition cost of $20,831 for 30 tablets. Takeda offers $0 copay assistance for those with commercial insurance, as well as patient assistance for patient who don’t have insurance or whose insurance doesn’t cover Iclusig.

The newest approval in newly diagnosed patients with Ph+ ALL was supported by data from the phase 3 PhALLCON study, which met the primary endpoint of minimal residual disease (MRD)-negative complete remission.

MRD-negative complete remission is a composite endpoint that reflects molecular and clinical responses. Doctors use MRD to measure the effectiveness of a treatment and predict who is at risk of disease relapse. In a clinical trial, MRD is a considered a surrogate endpoint. Takeda is required to conduct a confirmatory trial.

In the study, patients received either Inclusig or imatinib plus reduced-intensity chemotherapy. Patients receiving Inclusig achieved a greater than two-fold improvement in the rate minimal residual disease compared with imatinib. No new safety signals were identified.

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