FDA Approval Alert: Iclusig Granted Full Approval for CML
The US Food and Drug Administration has granted Iclusig® (ponatinib; ARIAD) full approval for the treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) for whom no other tyrosine kinase inhibitor (TKI) therapy is indicated; and for the treatment of adult patients with T315I-positive CML (chronic phase, accelerated phase, or blast phase) or T315I positive Ph+ ALL.
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The drug was initially approved in December 2012 under the FDA’s accelerated approval program, which prioritizes promising therapies that treat serious conditions by using surrogate endpoints while the drug developer continues to conduct additional studies to confirm clinical benefit.
This full approval was based on data from the 4-year PACE trial, which was presented at the American Society of Clinical Oncology and European Hematology Association 2016 meetings. The trial included 449 patients with CML and Ph+ ALL or T315I mutation, all of whom received ponatinib at a starting dose of 45 mg/day. Patients who received the drug demonstrated improved response.
“The data on Iclusig continue to show that with a minimum follow-up of 48 months, many chronic phase CML patients in the PACE trial have retained long-term cytogenetic and molecular responses,” stated Timothy P Clackson, PhD, president of research and development and chief scientific officer at ARIAD. “We are pleased to have received full approval of this medicine that was discovered and developed by ARIAD scientists to address rare cancers for patients who may have no other targeted treatment option.”
The most common any-grade, treatment-related adverse events associated with ponatinib were hypertension (69%), rash (63%), abdominal pain (48%), fatigue (47%), headache (43%), arterial ischemia (42%), dry skin (42%), constipation (41%), arthralgia (32%), nausea (28%), pyrexia (26%), peripheral neuropathy (24%), myalgia (24%), pain in extremity (23%), back pain (21%), diarrhea (20%).
“The four-year follow-up and updated safety profile demonstrate durability of responses in this heavily pre-treated population,” commented Jorge Cortes, M.D., professor and deputy chair, Department of Leukemia, The University of Texas MD Anderson Cancer Center, and a leading investigator in the PACE trial. “These results solidify ponatinib as an important and valuable treatment option for refractory patients with CML where no other TKI therapy is appropriate, including those who have the T315I mutation.”
ARIAD also stated that is will now begin enrolling patients in a post-marketing trial of ponatinib, which is expected to inform the optimal dosing of the drug. Patients with CP-CML who are resistant to at least two approved TKIs will be randomly assigned to receive once-daily administration of 45 mg (cohort A), 30 mg (cohort B) or 15 mg (cohort C) of ponatinib. Investigators are confident this will help to further improve outcomes.--Sean McGuire
