Genzyme Receives FDA Letter (NASDAQ:GENZ)

Genzyme Corporation has announced that they have received a complete response letter from the Food and Drug Administration about the company’s supplemental New Drug Application for Clolar in patients suffering from acute myeloid leukemia or untreated older patients with AML. In the response letter to Genzyme Corporation, FDA has clearly mentioned that they need to have a randomized and controlled clinical trial be conducted for label expansion of Clolar. Genzyme’s Clolar drug is currently approved for pediatric ALL patients who have relapsed or have refractory diseases. FDA has also granted six months of extended market exclusivity to Clolar under the Best Pharmaceuticals for Children Act.

In September, FDA Oncologic Drugs Advisory Committee voted for Clolar and had emphasized that it was necessary to establish the efficacy and safety of Clolar for the proposed adult AML indication. Genzyme had then sought of approving the expansion of indication based on the findings from a single-arm trial in an adult AML population. However, now Genzyme is deciding to have meeting with the FDA to discuss the optimal path forward and what they are studying to make sure they satisfy the regulatory requirements. Currently, Genzyme is conducting a randomized, placebo-controlled Phase 3 trial in relapsed and refractory adult AML patients.