Genzyme Fails to Receive Approval for Leukemia Drug

On Wednesday, Genzyme Corp. (GENZ) failed to receive U.S. Food and Drug Administration (FDA) approval for Clolar in elderly adults with acute myeloid leukemia (AML). Clolar is currently approved for pediatric acute lymphoblasic leukemia (ALL) patients who have relapsed or refractory disease after at least two prior regimens.

Genzyme was looking to get Clolar approved for the treatment of previously untreated adults aged 60 or older with AML, who have at least one unfavorable prognostic risk factor.

In its Complete Response Letter (CRL), the agency stated that Genzyme will have to conduct a randomized, controlled study to gain approval. The response does not come as a surprise as it is in-line with the recommendation of the agency’s Oncologic Drugs Advisory Committee.

In early Sep 2009, the FDA’s Oncologic Drugs Advisory Committee voted (9-3) that Genzyme needs to conduct a randomized controlled study in order to gain approval for a label expansion for Clolar.

Although the committee found that the single-arm study results submitted by the company showed that Clolar was an active agent in acute AML patients, the committee stated that a randomized study would be required to better establish Clolar’s efficacy and safety in the proposed patient population.

Genzyme is currently conducting a randomized phase III trial comparing Clolar in combination with cytarabine to cytarabine alone in relapsed and refractory adult AML patients 55 years old or older. Results should be out in 2011.

Genzyme intends to meet with the FDA to discuss the regulatory path forward. With the delay in Clolar’s approval being expected, we do not expect the news to have a major impact on Genzyme’s shares.

We currently have an Underperform rating on Genzyme, which is facing problems due to contamination issues at its Allston manufacturing plant.