December 16, 2012
FDA Approves New Drug For Treatment Of Rare Leukemia
redOrbit Staff & Wire Reports - Your Universe OnlinePonatinib, a third-generation, multi-targeted tyrosine kinase inhibitor that will be marketed by Ariad Pharmaceuticals under the brand-name Iclusig, received the go-ahead on Friday, some three months before the agency was required to act on its application, said MedPage Today Senior Editor John Gever.
In addition to being used to treat CML, it was also approved for use in adults suffering from Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), Gever added. The approval was based on a single phase II trial, in which researchers reported "rates of major cytogenetic and hematologic responses to daily oral treatment with ponatinib ranged from 33% to 70%, depending on the disease subtype."
"Toxicities were generally mild, except for a few patients who experienced serious pancreatitis," he added.
Ponatinib's approval had been expected, though the product's label allows for broader use than some experts had anticipated, Andrew Pollack of the New York Times reported on Friday. Ariad's Iclusig is to be taken in tablet form once per day and will carry an annual cost of approximately $115,000.
“The approval of Iclusig is important because it provides a treatment option to patients with CML who are not responding to other drugs,” Dr. Richard Pazdur, director of the FDA's office of cancer drugs, said in a statement, according to Pollack. He added that the pharmaceutical company estimates that as many of 2,500 CML patients each year wind up seeking new treatment options, making them candidates for the newly approved medication.
Iclusig will carry a black box warning, which states that use of the drug could lead to liver toxicity and arterial thrombosis, Bloomberg News noted. As a result of that advisory, Ariad's stocks experienced their largest one-day decline since October 2008, falling 21% to $18.93 at the close of business Friday.
Cowen Group analyst Phil Nadeau told the news organization that investors "were surprised and disappointed" by the warning, which they believe could limit the use of ponatinib. In a research note, Nadeau said that he believes the health concerns might be exaggerated, and that physicians have called the drug "very well-tolerated." Nonetheless, the company hailed the agency's approval as a victory.
"Today´s FDA approval of Iclusig is an important advance in the treatment of patients with CML and Ph+ ALL who are resistant or intolerant to prior TKI therapy," Dr. Harvey J. Berger, chairman and CEO of ARIAD, said in a statement. "Within less than five years, we were able to bring Iclusig from the start of clinical development to U.S. approval, achieving a major milestone in ARIAD´s history. We have now transformed ARIAD into a commercial oncology company addressing major unmet medical needs for cancer patients."