The U.S. Food and Drug Administration (FDA) has granted full approval to ponatinib (Iclusig) for the treatment of chronic phase, accelerated phase, or blast phase chronic myeloid leukemia in chronic phase (CP-CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) for adult patients not indicated for any other tyrosine kinase inhibitor (TKI) therapy, according to a release.1
Manufactured by Ariad Pharmaceuticals, ponatinib was evaluated in the phase 2 PACE (ClinicalTrials.gov Identifier: NCT01207440) trial, which evaluated 4-year follow-up data of 449 patients with CML or Ph+ ALL who were resistant or intolerant to dasatinib or nilotinib.
About 93% of observed patients received at least 2 approved TKIs and 56% received at least 3 approved TKIs prior to treatment.
In total, 55% of the patients had achieved the primary endpoint of major cytogenetic response at any time during the study, while 39% of patients achieved major molecular response at any time.
About one-third of patients (150 of 449) experienced arterial occlusive events with 4 years of follow-up, including 21% with cardiac vascular, 12% with peripheral vascular, and 9% with cerebrovascular arterial occlusive.
Ponatinib is also being evaluated for optimal dose through the Optimizing Ponatinib Treatment in CML (OPTIC; ClinicalTrials.gov Identifier: NCT02467270) randomized trial, which is enrolling patients with CP-CML who are resistant to at least 2 approved TKI therapies. Initial data are expected at the American Society of Hematology 2017 meeting.
- 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. Ariad website. http://investor.ariad.com/phoenix.zhtml?c=118422&p=irol-newsArticle&ID=2226080. Updated November 29, 2016. Accessed November 30, 2016.