Genzyme Corp.’s (GENZ) Clolar could face a delay in receiving approval for an expanded indication. The drug will be coming up for review before the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) on Sept 1 and questions have been raised regarding the lack of a randomized study supporting the application.

The company is seeking to get Clolar approved for the treatment of previously untreated adults aged 60 or older with acute myeloid leukemia (AML) who have at least one unfavorable prognostic risk factor. The company is seeking approval on the basis of a phase II study in which Clolar was evaluated in elderly patients with previously untreated AML and at least one other factor that gave them a poor prognosis. The study did not include any comparison with a placebo.

Clolar is currently approved for pediatric acute lymphoblastic leukemia (ALL) patients who have relapsed or refractory disease after at least two prior regimens. The new indication would expand the patient base significantly for Clolar. According to the American Cancer Society, approximately 12,810 people will be diagnosed with AML in the United States in 2009 and about 70% of these patients will die from the disease.

At the upcoming meeting, the Committee will decide whether another study will be needed to approve Clolar for the expanded indication. The Committee will also evaluate whether the patients studied by Genzyme were not suitable for chemotherapy.

We believe the committee will ask the company to conduct a randomized study in order to gain approval for the expanded indication. We note that Johnson & Johnson’s (JNJ) cancer drug, Zarnestra, met a similar fate in 2005 when the FDA’s oncology committee voted against approving the drug based on data from a single-arm phase II study.  

In fact, in 2007, the agency had recommended that Genzyme should conduct a randomized controlled study in which patients receiving Clolar would be compared with patients receiving placebo.

Meanwhile, the FDA action date for Clolar has been pushed back to December. A delay in Clolar’s approval would be a setback for the company, which is already facing problems due to contamination issues at its Allston manufacturing plant. We have an Underperform rating on Genzyme.

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