Doctors Worry Leukemia Drug May Be Banned by FDA

The U.S. Food and Drug Administration (FDA) announced this week that sales of the leukemia drug, Iclusig, could be suspended due to potential risks.

For some patients diagnosed with chronic myeloid leukemia (CML), this risky drug may be the only chance they have for survival.

CML is a cancer that starts inside the bone marrow. This is the soft tissue in the center of bones that helps form all blood cells. CML causes an uncontrolled growth of immature cells that make a certain type of white blood cell called myeloid cells.

The issue at the heart of this decision by the FDA to halt sales of Iclusig was that it significantly raises the odds of heart attacks, strokes, blindness, life-threatening blood clots, death and amputations.

However, despite these increased risks, many doctors who are treating CML believe that nothing else has worked for their patients and that their lives are dependent upon this drug.

“My concern is that I have patients right now who are benefiting from this medication with very few side effects, and if they’re on the end of a one-month prescription, what’s going to happen when their medication runs out?” said Dr. Brian Druker, the director of the Knight Cancer Institute at Oregon Health and Science University. “Without this medication, they won’t have long to live. My patients are panicked.”

The FDA has assured that patients who need the drug will still be able to obtain it, but that doctors will have to file applications for each patient, a process that some say can be tedious and can leave patients caught up in red tape, while  having no medication.

Dr. Michael Mauro, a leukemia specialist at Memorial Sloan-Kettering Cancer Center in New York, said 23 leukemia specialists and three patient advocacy groups had sent the drug agency a letter stating that they were concerned that the sudden withdrawal of the drug would interrupt treatment for patients with no other good options.

Iclusig, also called ponatinib, was approved in December 2012 to treat patients who had CML that did not respond to other drugs, as well as another rare type of leukemia, CGL. The disease is a relatively uncommon form of leukemia, with about 5,000 cases a year and 600 deaths in the United States. Iclusig is a daily pill and Ariad’s only approved product. The wholesale price of the drug was $115,000 a year. This drug brought in $13.9 million in sales in the second quarter.

Iclusig was given accelerated approval by the FDA under a program that allows some important drugs to reach the market quickly, without the full evidence of safety and efficacy that is usually required, subject to further studies to confirm the drug’s benefit.

CML had been treated with a class of drugs that led to dramatically improved long term survival rates (95.2 percent) since being introduced in 2001, the first of which was called Gleevec. It is believed that these drugs have revolutionized treatment of CML and allow most patients a good quality of life when compared to the former chemotherapy drugs. However, some patients did not respond to Gleevec or became resistant to it. Iclusig has become their only hope.

As with most drugs, there are side effects or adverse reactions that sometimes outweigh the benefits. Iclusig not only fights the cancer cells, but also carries serious cardiovascular dangers.

In a bulletin Thursday, the FDA said that 48 percent of patients treated with the drug in an early-stage trial and 24 percent of patients taking Iclusig in a mid-stage trial have suffered “serious adverse vascular events.” Those figures are unusually high, and higher than what was reported from the initial studies done before the drug was approved.

Some of the reported cases involved blockages in blood vessels, which led to heart attacks, strokes, blindness and lack of blood flow to the extremities. Some of the patients were only in their 20s, many of whom had no risk factors for cardiac disease. The FDA said that some patients’ reported serious side effects as early as two weeks after starting the drug.

Stephanie Yao, FDA spokeswoman, said that studies of the drug to date, which involved 530 patients, reported at least 14 deaths related to cardiovascular issues.

Doctors who specialize in the treatment of CML worry that patients will suffer if this drug is unavailable. “What we don’t want to have happen is for a good drug, ponatinib, to be killed and not given to patients,” Dr. Javid Moslehi of Dana-Farber Cancer Institute and Brigham and Women’s Hospital in Boston said.

The setbacks for the drug have really shaken up the Cambridge, Massachusetts drug maker, Ariad Pharmaceuticals. Stock lost 85 percent of its value since October 9th, when it first announced that the FDA had halted further enrollment in clinical trials of Iclusig due to safety concerns.

For more information, contact one of our Gacovino Lake attorneys at 1-800-246-HURT (4878).

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