Advocates Lobby for Continued Access to Ponatinib in US

Roxanne Nelson

November 26, 2013

The leukemia drug ponatinib (Iclusig) was temporarily pulled from the market in the United States last month by its manufacturer, Ariad Pharmaceuticals, at the request of the US Food and Drug Administration (FDA). The reason cited was safety concerns; evolving data have shown an increased incidence of serious cardiovascular toxicities.

After reviewing the same data, European authorities decided that ponatinib could remain on the market, but with strengthened warnings.

Within a week of the drug being removed from the American market, Ariad Pharmaceuticals laid off 40% of its workforce in the United States and its stock price plunged.

Even though ponatinib remains available in Europe, Ariad is no longer being paid to distribute it in the United States under the FDA emergency program.

This has led to active lobbying from patients and physicians for continued access to the drug, according to a report in the Boston Globe.

The day after the drug was withdrawn, 23 physicians signed a letter to Richard Pazdur, MD, director of the hematology and oncology products office at the FDA Center for Drug Evaluation and Research, asking the agency to speed up a process that would allow patients who were already using ponatinib to keep receiving it. The program went into effect within 3 days.

This seemingly successful lobbying effort underscores the growing input of patients, physicians, and other advocates in making decisions about drug accessibility, according to the Boston Globe report. The view is that physicians should be allowed to prescribe drugs with serious adverse effects to patients willing to take that risk because they generally have few or no other options.

"We understand the need for access to products for the treatment of serious and life-threatening diseases and that there are some patients who are currently receiving [ponatinib] who will require continued drug access," Dr. Pazdur wrote in response to the physicians' letter.

Ponatinib is indicated for chronic-phase, accelerated-phase, and blast-phase chronic myeloid leukemia (CML), and for Philadelphia chromosome-positive acute lymphoblastic leukemia in patients who do not benefit from other therapies, as previously reported by Medscape Medical News. Although there are several other drugs now available to treat CML, ponatinib is the only one that is active against the threonine-to-isoleucine mutation at position 315 (T315I), which is present in up to 20% of patients who exhibit resistance to tyrosine kinase inhibitors.

For that reason, despite the real risks associated with this drug, it is the only alternative for a subgroup of patients. According to the Boston Globe, within hours of the drug being pulled from the market on October 31, loosely organized groups of physicians and patients began making calls and writing to regulators to make sure that those already taking ponatinib would not lose access.

"We support the FDA and the job they have to do," said Hans Loland, a computer systems analyst from Woodinville, Washington, who organized an Internet group for CML patients. "But we want a balanced approach."

In the Boston Globe report, he noted that "our concern is the longer this is dragged out, there are patients who are going to lose access to the drug and there are doctors who may not prescribe it to patients who could benefit."

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