Agency is asking for a randomized, placebo-controlled trial to gain approval in adult AML.

FDA has recommended that Genzyme perform an additional clinical study with its cancer medication Clolar for label expansion into previously untreated older adult patients with acute myeloid leukemia (AML) and at least one unfavorable baseline prognostic factor. Clolar is currently approved for pediatric acute lymphoblasic leukemia (ALL) patients who have relapsed or have refractory disease after at least two prior regimens.

It has orphan drug designation for AML and pediatric ALL and seven years of market exclusivity in the U.S. for the approved indication. The FDA granted six months of extended market exclusivity to Clolar under the Best Pharmaceuticals for Children Act.

Genzyme had sought approval to expand the indication based on the findings from a single-arm trial in an adult AML population. The FDA is now asking for a randomized, placebo-controlled trial, and the company will meet with the agency to discuss the optimal path forward, including what additional or ongoing studies may satisfy regulatory requirements.

Genzyme is currently conducting a randomized, placebo-controlled Phase III study comparing Clolar in combination with cytarabine to cytarabine alone in relapsed and refractory adult AML patients 55 years old or older. Results are expected in 2011.

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