HealthDay News — The FDA has approved ponatinib (Iclusig) to treat two drug-resistant forms of leukemia, chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), in adults.
The drug’s approval comes three months before the agency’s official deadline for acting on the marketing application, and was based on results from a single phase II trial presented at the American Society of Hematology’s annual meeting.
The PACE trial included 449 patients with either CML or Ph+ ALL, and demonstrated major cytogenetic and hematologic responses with ponatinib ranging from 33% to 70%, depending on the disease subtype. Toxicities were generally mild, except for a few patients who experienced serious pancreatitis.
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Response rates in the PACE trial were highest in patients with chronic- and acute-phase CML, ranging from 50% to 70%. For patients with blast-phase CML or Ph+ ALL, response rates were in the 33% to 35% range.
Ponatinib is a third-generation tyrosine kinase inhibitor. All patients in the trial had previously failed therapy with other approved tyrosine kinases, showing resistance or intolerance to dasatinib (Sprycel) or nilotinib (Tasigna).
“The approval of Iclusig is important because it provides a treatment option to patients with CML who are not responding to other drugs, particularly those with the T315I mutation who have had few therapeutic options,” Richard Pazdur, MD, director of the FDA’s Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research, said in a press release.
Common side effects included high blood pressure, rash, abdominal pain, fatigue, headache, dry skin, constipation, fever, joint pain and nausea. The product’s label will include a boxed warning that users are at heightened risk of blood clots and liver poisoning.
Iclusig is marketed by Ariad Pharmaceuticals, based in Cambridge, Mass.
