The agency declined to approve decitabine for use in elderly patients with acute myelogenous leukemia because the pivotal study failed to demonstrate a statistically significant survival benefit in the pre-specified primary analysis. FDA’s “complete response” letter tracks with a negative evaluation by the Oncologic Drugs Advisory Committee in February.

The agency declined to approve decitabine for use in elderly patients with acute myelogenous leukemia, pointing to the failure of the pivotal trial to show a survival benefit on primary analysis. FDA’s “complete response” letter tracks with a negative evaluation by the Oncologic Drugs Advisory Committee in February.

Cyclacel will submit a Special Protocol Assessment request for its Phase III trial of sapecitabine in acute myeloid leukemia, as well as for a separate trial in myelodysplastic syndromes, during the first quarter of 2010. The company announced Dec. 15 that at a recent type A meeting with FDA, the agency confirmed that the proposed design for a randomized study would be acceptable for the SPA process - through which sponsors gain insight from FDA about clinical trial design and regulatory expectations. Consulting with FDA on the Phase III program is an even more important step in this space following an advisory committee review of two competitors; during its Sept. 1 review of applications for Genzyme's Clolar and Vion's Onrigin for the niche indications to treat elderly patients with AML, FDA's Oncologic Drugs Advisory Committee determined that randomized trials, though difficult to conduct in the space, are necessary for approval (1"The Pink Sheet," Sept. 7, 2009). Both applications have since received "complete response" letters from FDA requesting randomized, controlled trials. Genzyme has already initiated such a study (2Pharmaceutical Approvals Monthly, October 2009). Vion, however, may not be able to fulfill the request (3"The Pink Sheet" DAILY, Dec. 14, 2009)