Clinical Study of Leukemia Drug Clolar Under Fire at FDA

Officials at the Food and Drug Administration are taking aim at a clinical study meant to support broader uses of the Genzyme Corp. leukemia drug Clolar.

Genzyme wants Clolar, which is approved for treating a form of leukemia in children, permitted to treat adults with acute myeloid leukemia (AML), a life-threatening blood cancer. But serious questions about the credibility of a key clinical study in support of the drug have thrown a wrench in the works, according to a Reuters news report.

“The lack of a randomized study combined with the heterogeneous patient population regarding AML prognostic factors makes interpretation of the study results difficult,” FDA staffers said in a memo released today before an advisory panel meeting set for next week.

The clinical trial of Clolar tested the drug in elderly patients who had a poor prognosis as a result of untreated AML and one or more other factors. However, there was no control group in the study to test those patients against a group of patients not treated with the drug, the FDA said.

The FDA had told Genzyme in 2007 that a randomized controlled study of Clolar should compare patients treated with the drug to others not treated, but the company’s trial did not do so. The FDA recently extended its deadline for a decision on Clolar until December 2009, the company said

Advisory Panel Set to Meet

Next week, the FDA staff will ask its advisory panel of medical experts whether the trial Genzyme conducted for Clolar is sufficient to determine the drug’s risks and benefits and decide whether to grant broader approvals for the drug. The FDA usually follows the recommendations of its advisory panels, even though it is not required to do so. A final decision on Clolar’s future is expected sometime after the advisory panel meeting.

Genzyme said its study of Clolar produced data which “demonstrate clinically meaningful benefits and manageable risks” of the drug. The study found that 45 percent of AML patients had their leukemia go into remission, the company said. Clolar, known generically as clofarabine, is delivered via intravenous injection.

Annual sales revenues Clolar are projected to reach $150 million in 2013 and $240 million by 2015, the Reuters report said. More than 12,000 Americans will be diagnosed with AML this year and nearly 70 percent of those diagnosed will die from the disease, Genzyme said.

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