Leukemia drug Iclusig gets approved: Possible PR lead

Leukemia drug Iclusig gets approved: Possible PR lead

Steve’s breakdown: With a drug costing more than $100K/year wholesale, our prediction is this drug needs more PR than is does advertising.

CAMBRIDGE, MA: First there was Gleevec, the wonder cancer drug. Then came the sons of Gleevec. Now there is the grandson of Gleevec.

The Food and Drug Administration on Friday approved another Gleevec-like drug for the treatment of chronic myeloid leukemia, or C.M.L., adding to a crowded field vying to treat this rare cancer of white blood cells.

The new drug, Iclusig from Ariad Pharmaceuticals, works in some patients not helped by Gleevec or some of its imitators.

“The approval of Iclusig is important because it provides a treatment option to patients with C.M.L. who are not responding to other drugs,” Dr. Richard Pazdur, director of the agency’s office of cancer drugs, said in a statement.

Iclusig, known generically as ponatinib, is the first product to reach the market for Ariad, a biotechnology company in Cambridge, Mass., that was founded in 1991.

A tablet taken once a day, Iclusig (pronounced eye-CLUE-sig) will have a wholesale price of about $115,000 a year, the latest cancer drug to pierce the $100,000-a-year level. Ariad said the price was about 15 percent higher than the drug’s competitors’.

While the approval was expected, it came three months before the federal agency’s deadline of March 27, and the drug’s label allows broader use of the drug than some analysts expected. However, the label also contains a boxed warning about the side effects of blood clots and liver toxicity, something analysts did not expect.

Ariad’s shares, which have roughly doubled in the last year, sank 21 percent to $18.93 on Friday.

Chronic myeloid leukemia would seem at first glance to be an unattractive target for pharmaceutical companies. Not only is it rare — with about 5,000 new cases a year in the United States and 600 deaths — it is also one of the more effectively treated of cancers.

But it is also a well-understood cancer. Drug companies know how to attack it, so they do — something akin to looking for lost keys under the lamppost because that is where the light is. Iclusig is the third drug for chronic myeloid leukemia approved by the agency this year, after Pfizer’s Bosulif and Teva’s Synribo.

The disease occurs when pieces of two separate chromosomes come together to form what is known as the Philadelphia chromosome. A fusion of genes creates an aberrant protein, known as BCR-ABL, which fuels uncontrolled cell growth.

Gleevec, developed by Novartis and approved in 2001, inhibits the action of that errant protein. It turned chronic myeloid leukemia from a death sentence into a chronic disease for many patients and is still considered the paragon of molecularly targeted drugs. Global sales were $4.7 billion in 2011.

But about 20 to 30 percent of patients have cancers that do not respond to Gleevec or that develop resistance to it.

So companies developed other drugs — Tasigna from Novartis and Sprycel from Bristol-Myers Squibb — that also inhibit the aberrant protein but work in many of the cases that are resistant to Gleevec. But some cases do not respond to those drugs, either.

Iclusig was designed by chemists and computers at Ariad to work against cancer cells with a mutation — known as T315I — that makes them resistant to Gleevec, Tasigna and Sprycel.

However, Iclusig was approved not only for patients with that mutation but for any patient who has tried one of the other three drugs without success. Ariad estimates that each year about 2,500 patients with the disease switch therapies, making them candidates for Iclusig.

That can still be a lucrative market, given the high price of the drugs and the fact that patients must take them for the rest of their lives, which is usually many years. Dr. Harvey J. Berger, chief executive of Ariad since the company’s inception, said he expected annual sales to reach $600 million to $800 million in a few years.

The F.D.A. approved the drug based on a trial with 449 patients, all of whom received Iclusig. The patients had already tried treatment with one, two or three of the other drugs.

About 54 percent of patients in the early stage of the disease had a major cytogenic response, meaning a reduction in cells with the Philadelphia chromosome.

Iclusig was also approved Friday as a treatment for an even rarer cancer, acute lymphoblastic leukemia characterized by presence of the Philadelphia chromosome.


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