Ariad Pharmaceuticals Inc fka ARIA

[LUMA14]

The Slippery Slope: Risks and Confusion

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Ponatinib (Iclusig), a $9,200-a-month leukemia drug that also has not been shown to lengthen lives. The FDA required a black box warning because clots formed in at least 27% of patients leading to possible heart attacks and strokes, potentially fatal heart failure, and potentially fatal liver failure.
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But patients can manage their symptoms by adjusting the dose. In addition, they can remain on the drug for a longer period of time than alternative treatments because of a progression-free survival benefit, the company said.

Ariad Pharmaceuticals, maker of the leukemia drug ponatinib, is a small company with just one drug on the market and a limited group of potential patients.

Only about 1,300 Americans a year, and 3,000 to 4,000 people worldwide, are candidates for the drug, said Frank Haluska, MD PhD, chief medical officer of the company, based in Cambridge, Mass.

Haluska acknowledged the drug's lack of proven survival and quality-of-life benefits and the problem with using surrogates as a basis for approval. But, he said, "all surrogates are not created equal."

For leukemia, he said, surrogates are better indicators of such benefits than with some other cancers.

Prior to approval of the drug, about 50% of people with the type of leukemia treated by ponatinib survived 2 years, he said. The company's experience with the drug so far indicates that 86% are surviving to 2 years.

In the years to come, Haluska said, the company will be studying survival as well as whether dosing can be changed to reduce serious complications.

Paul Kluetz, MD, acting deputy director of the FDA's Office of Hematology and Oncology Products, said there is a trade-off between the robust data that prove a survival benefit and quickly getting drugs on the market after they have showed some antitumor activity.

"It's a risk we accept for hastening the delivery of promising agents to cancer patients," he said.

Risk versus Benefit
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link to cancer drugs an monthly prices