The drug is a tyrosine kinase inhibitor, meant as a successor to drugs like Bristol-Myers’ dasatinib (Sprycel), which reached the market six years ago. The FDA approved the drug for patients with all forms of chronic myeloid leukemia or Philadelphia chromosome-positive acute lymphoblastic leukemia who are resistant or unable to tolerate other tyrosine kinase therapies already on the market.
On Monday Ariad, based in Cambridge, MA, reported at the American Society of Hematology annual meeting in Atlanta that, in a key clinical trial, 56 percent of patients on ponatinib for 12 months who no longer responded to other treatments had a major response—impressive results in patients this sick. The FDA said it based its approval on the results of that trial.
“The approval of Iclusig is important because it provides a treatment option to patients with [chronic myeloid leukemia] who are not responding to other drugs,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release.
Ariad CEO Harvey Berger said in a webcast after the approval was announced that the drug will be available in about two weeks from specialty pharmacies, and will cost $115,000 a year per patient at the wholesale price. The company owns all rights to ponatinib, and Berger said it is already fully able to market and manufacture the drug to meet demand. He also said that the company plans now to test the drug in gastrointestinal stromal tumors, a rare form of stomach cancer.
The approval is a nice comeback after the FDA rejected another Ariad drug in June, ridaforolimus (Taltorvic) for sarcomas, which it was developing jointly with Merck (NYSE: MRK), based in Whitehouse Station, NJ.
Approximately 5,000 new cases of chronic myeloid leukemia are diagnosed annually in the U.S. Ponatinib is particularly active against a particular cellular mutation, T315I, that is found in about 10 percent of patients and makes them resistant to other approved tyrosine kinase inhibitors.
Ponatinib was approved with a black box warning cautioning patients and physicians that the drug can cause blood clots and liver toxicity, but company officials said in the webcast that they do not expect the warning to have any impact on sales of the drug.