Celgene Corporation's Vidaza (azacitidine) has received expanded US Food and Drug Administration (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 US 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 of the University 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 Avidaza 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 per cent 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 leukaemia (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 per cent compared to 26.2 per cent for CCR. Patients treated with Vidaza received treatment for a median of nine cycles.

"The clinical data from this randomized phase-III controlled study demonstrated that patients with higher-risk MDS treated with Vidaza benefit from a significant survival advantage, a critical measure of a drug's effectiveness," said Lewis Silverman of the Mount Sinai Medical Center in New York City. Dr Silverman was the lead author and principal investigator for the original Vidaza approval study (CALGB 9221) and an author and investigator of the international AZA-001 survival trial. "Additionally, the efficacy and safety profile of Vidaza allows for long-term therapy in patients with higher-risk MDS, underscoring the ability to treat until disease progression for optimal survival benefit."

In the AZA-001 study, the most commonly occurring adverse reactions for patients with higher-risk MDS receiving Vidaza were thrombocytopenia (69.7 per cent), neutropenia (65.7 per cent) and anaemia (51.4 per cent).

"This decision by the FDA reflects the unprecedented survival advantage demonstrated by VIDAZA in patients with higher risk MDS," said Mohamad A Hussein, global head, Medical Affairs, Hematology of Celgene, formerly of the H Lee Moffitt Cancer Center and Research Institute. "VIDAZA is another example of Celgene developing novel therapies for critical blood diseases that are enabling patients to live for years, rather than weeks and months. Today's decision strengthens our Company's ability to deliver VIDAZA and our other therapies to patients in need around the world."


In May 2004, Vidaza became the first drug approved by the FDA for the treatment of patients with Myelodysplastic Syndromes (MDS). The FDA approved Vidaza, the first in a new class of drugs called demethylation agents, for treatment of all five MDS subtypes, which include both low-risk and high-risk patients.

Vidaza is an epigenetic compound believed to exert antineoplastic effects by causing hypomethylation of DNA and direct cytotoxicity on abnormal haematopoietic cells in the bone marrow.

Myelodysplastic syndromes (MDS) are a group of hematologic malignancies that affect approximately 300,000 people worldwide. Myelodysplastic syndromes occur when blood cells remain in an immature or 'blast' stage within the bone marrow and never develop into mature cells capable of performing their necessary functions.

Celgene Corporation, based in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of novel therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation.