Vidaza (azacitidine) of Celgene Corporation received expanded U.S. Food and Drug Administration (US FDA) approval to reflect new overall survival achieved in the AZA-001 survival study of patients with higher-risk myelodysplastic syndromes (MDS).

This expanded indication supplements the 2004 FDA authorization of Vidaza as the first therapy approved in the U.S. for the treatment of patients with all five French American British (FAB) subtypes of MDS. Vidaza is also the first and only drug to show a statistically significant and clinically meaningful extension of survival in higher-risk MDS patients.

The overall survival detailed in the expanded FDA approval of Vidaza is extremely important for patients with higher-risk MDS, a group with limited options and median survival of about 15 months with classical treatments," said Pierre Fenaux, M.D., Ph.D. of the Universite of Paris and lead investigator of the AZA-011 survival trial. "Vidaza, however, is also effective across a broad range of MDS subgroups, including WHO-classified AML patients, the largest subgroup in our study."

The approval is based upon the significant improvement in overall survival achieved in the Vidaza survival trial (AZA-001), the largest, international randomized Phase III controlled study ever conducted in higher-risk MDS. The median overall survival for patients treated with Vidaza in the study was 24.5 months compared to 15 months for conventional care regimens (CCR), demonstrating a survival benefit of over 9 additional months with a stratified log-rank p-value of 0.0001. The hazard ratio describing this treatment effect was 0.58 (95 percent confidence interval of 0.43 to 0.77). The extension of survival was seen across the relevant patient subgroups including those greater than 65 years, as well as poorer prognostic groups such as those with World Health Organization (WHO) classified acute myelogenous leukemia (AML), which formed 31 percent of the enrolled patients, and patients with poor risk cytogenics. In the trial, the two-year survival rate for patients with higher-risk MDS treated with Vidaza was almost doubled with 50.8 percent compared to 26.2 percent for CCR. Patients treated with Vidaza received treatment for a median of nine cycles.