Dec 10 2008
Celgene International Sàrl has announced results from a subset analysis of the international phase III trial (AZA-001) demonstrating that the overall survival benefit observed in higher-risk MDS patients extended to patients with acute myeloid leukemia (AML).
The data, presented at the 50th Annual Meeting of the American Society of Hematology (ASH), reported that patients with WHO-defined AML who were treated with VIDAZA (azacitidine) achieved significantly improved overall survival compared to those treated with a conventional care regimen (CCR).
The AZA-001 trial recently showed that treatment with VIDAZA provides an unprecedented survival benefit in patients with higher-risk myelodysplastic syndromes (MDS). Based on these results, VIDAZA received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for certain patients with higher-risk MDS and WHO-defined AML and gained an approval from the U.S. Food and Drug Administration for an expanded label to include the survival data in higher-risk MDS.
Almost one third of the patients (113 of 358) enrolled in the AZA-001 study met the WHO criteria for AML (median 23% bone marrow blasts), which has a poor prognosis and does not respond well to conventional chemotherapy. This subset analysis of the AZA-001 study showed the median overall survival was 24.5 months with VIDAZA compared to 16.0 months with CCR (p=0.005). Additionally, 50 percent of the AML patients who were treated with VIDAZA survived at least two years, compared to only 16 percent of AML patients treated with CCR.
The subset analysis also showed that patients treated with VIDAZA had fewer infections requiring intravenous antibiotics and reduced rates of hospitalization and red blood cell transfusions.
"Patients with AML have an extremely poor prognosis," said Pierre Fenaux, M.D., of the University of Paris and lead investigator of the AZA-001 survival trial. "The results of this pivotal study show that the unprecedented survival benefit obtained by VIDAZA in higher-risk MDS extend to AML. I look forward to the benefits of this drug becoming available to patients in Europe and to further evaluation across all categories of AML."
In the AZA-001 study, the most commonly occurring adverse reactions were thrombocytopenia (69.7%), neutropenia (65.7%) and anemia (51.4%).