Celgene's Vidaza gets expanded US indication

US drugmaker Celgene says that Vidaza (azacitidine) has received expanded Food and Drug Administration approval to reflect new overall survival achieved in the AZA-001 study of patients with higher-risk myelodysplastic syndromes. This new indication supplements the 2004 authorization of Vidaza as the first therapy approved in the USA for the treatment of patients with all five French American British subtypes of MDS.

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, demonstrating a survival benefit of more than nine additional months with a stratified log-rank p-value of 0.0001.

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-classified acute myelogenous leukemia, which formed 31% 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% compared to 26.2% for CCR.