The Committee for Medicinal Products for Human Use (CHMP), which reviews applications for all 27 Member States in the European Union as well as Norway and Iceland, has recommended approval for azacitidine (Vidaza) for treatment of MDS and AML. The positive opinion includes important survival data from the AZA-001 trial in higher-risk MDS patients. The CHMP's opinion will be forwarded to the European Commission, which generally follows the recommendation of the CHMP and typically issues final marketing approval within two to three months.

Vidaza is the first and only medicinal product to demonstrate significantly extended survival for patients with higher risk myelodysplastic syndromes(MDS). MDS are a group of hematologic malignancies that affect approximately 300,000 people worldwide. MDS 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. Eventually, the bone marrow may be filled with blast cells suppressing normal cell development. According to current estimates, between 15,000 and 20,000 individuals are diagnosed each year with MDS in Europe.

"Vidaza is the first drug to meaningfully extend overall survival 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 Université de Paris and lead investigator of the AZA-001 survival trial. "Vidaza, moreover, is very effective across a broad range of MDS subgroups, including RAEB in transformation, now classified as AML by the WHO classification - one of the largest subgroups in our study."

The positive opinion from the CHMP was based upon safety and efficacy from clinical studies evaluating azacitidine in MDS-notably the significant improvement in overall survival achieved in the azacitidine 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 azacitidine in the study was 24.5 months compared to 15 months for conventional care regimens (CCR), demonstrating a survival benefit of over nine 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 all patient subgroups including those greater than 65 years, as well as poorer prognostic groups such as those with WHO classified acute AML, which formed over 30 percent of the enrolled patients, and patients with poor risk cytogenetics. The two-year survival rate for patients with higher-risk MDS treated with azacitidine was almost doubled with 50.8 percent compared vs. 26.2 percent for patients treated with conventional care regimens (CCR). Patients treated with Vidaza received treatment for a median duration of nine cycles.

Forty-five percent of the patients who were RBC transfusion-dependent at baseline, became RBC transfusion independent during the treatment period, compared with 11.4 percent of the patients in the combined CCR groups (a statistically significant (p < 0.0001) difference of 33.6 percent (95 percent CI: 22.4, 44.6). The median duration of RBC transfusion independence was 13 months for the patient treated by azacitidine.

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

"The CHMP recommendation is an especially important and positive milestone for Celgene. We are fully committed to deliver Vidaza to patients in need throughout the EU," said Philippe Van Holle, President of Celgene Europe. "We are optimistic that Vidaza will have broad support based on its value to patients and to the healthcare system. Upon approval we are prepared to initiate next steps for pricing, reimbursement and distribution plans for all EU member states."

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