Health Canada Approval of VIDAZA(R) Marks New Era of Treatment for Patients
With Rare Blood Cancers

Novel Blood Cancer Treatment Vidaza Almost Doubles Survival Rate For Patients With Myelodysplastic Syndromes (MDS) And Acute Myeloid Leukemia (AML)

OAKVILLE, ON, Dec. 10 /CNW/ - Health Canada has approved VIDAZA(R) (azacitidine), an important treatment option for advanced forms of a group of serious blood cancers. The approval of this treatment marks a major milestone in the ability to treat patients with higher-risk myelodysplastic syndromes (MDS) and a subset of acute myeloid leukemia (AML) who are not eligible for stem cell transplant. For the first time, patients now have real hope to significantly extend survival.

The pivotal clinical trial, AZA-001, published earlier this year in The Lancet Oncology, demonstrated that the two-year survival rate almost doubled with VIDAZA treatment (50.8 per cent) compared to conventional care regimens (26.2 per cent).(1) This data prompted the Joint Oncology Drug Review (JODR), the interprovincial body that reviews and makes listing recommendations for cancer drugs, to grant VIDAZA priority review status - only the second such designation to be granted this year.

VIDAZA represents a new generation of cancer treatments in Canada because it is the first drug that works via epigenetics to restore the normal function of genes that regulate cell growth and development. VIDAZA is the first proven treatment that alters the natural history of these diseases.

"This is indeed a great step forward and marks a paradigm change in the treatment of higher-risk MDS and AML," said Dr. Richard Wells , Director of the Crashley Myelodysplastic Syndrome Laboratory at Sunnybrook's Odette Cancer Centre in Toronto . "With VIDAZA we can, for the first time, offer patients a treatment that actively treats the disease and changes its natural course of progression and, as a result, offers real hope to live longer."

In addition to its unprecedented survival benefits, AZA-001 demonstrated that 45% of patients who were dependent on red blood cell transfusions at baseline in the VIDAZA group became transfusion independent compared to 11.4% in the conventional care group. The restoration of normal blood cell development liberates patients from the burden of transfusions and helps restore their quality of life.(1)

"This is tremendous news for patients and will have a remarkable impact on their quality of life" said Stan Chmelyk, President, Aplastic Anemia and Myelodysplasia Association of Canada (AAMAC). "All provinces must now move quickly to make this new option available as soon as possible through provincial drug plans."

VIDAZA will be commercially available in Canada in January 2010 .

Important Safety Information

    
    -   VIDAZA is contraindicated in patients with a known hypersensitivity
        to azacitidine or mannitol and in patients with advanced malignant
        hepatic tumours.
    -   In AZA-001, the most commonly occurring adverse reactions were
        thrombocytopenia (69.7%), neutropenia (65.7%), anemia (51.4%),
        constipation (50.3%), nausea (48.0%), injection site erythema
        (42.9%), and pyrexia (30.3%). The most commonly occurring Grade 3 or
        4 adverse reactions were neutropenia (61.1%), thrombocytopenia
        (58.3%), leucopenia (14.9%), anemia (13.7%) and febrile neutropenia
        (12.6%).
    -   Because treatment with VIDAZA is associated with anemia, neutropenia
        and thrombocytopenia, complete blood counts should be performed prior
        to initiation of therapy, and then as needed to monitor response and
        toxicity, but at a minimum, prior to each dosing cycle.
    -   Safety and effectiveness of VIDAZA in patients with MDS and hepatic
        impairment have not been studied as these patients were excluded from
        the clinical trials. VIDAZA is contraindicated in patients with
        advanced malignant hepatic tumors.
    -   VIDAZA has not been studied in patients with severe renal impairment.
        Patients with renal impairment should be closely monitored for
        toxicity since VIDAZA(R) and/or its metabolites are primarily
        excreted through the kidney.
    -   VIDAZA may cause fetal harm when administered to a pregnant woman.
        Females of childbearing potential should be apprised of the potential
        hazard to the fetus. Men should be advised not to father a child
        while receiving VIDAZA and for six months afterwards.
    -   Mothers should be advised not to breast feed while undergoing therapy
        with VIDAZA(R).
    

About MDS

MDS is a group of similar blood disorders that occur when blood cells in the bone marrow do not mature and eventually suppress normal cell development. An estimated 6,000 Canadians are living with MDS and approximately 1,500 new MDS cases are diagnosed each year.

Mean survival rates can range from approximately six to nine months for people with higher-risk MDS. For roughly 30 per cent of patients diagnosed with this disorder, the abnormal bone marrow cells eventually progress into acute myeloid leukemia (AML), a rapidly growing cancer of the bone marrow cells.

About VIDAZA

VIDAZA is approved by Health Canada for the treatment of adult patients with Intermediate-2 and High-risk myelodysplastic syndrome (MDS) and Acute Myeloid Leukemia (AML) with 20-30% blasts and multi-lineage dysplasia who are not eligible for hematopoietic stem cell transplantation.

VIDAZA is believed to exert its antineoplastic effects by causing hypomethylation of DNA and cytotoxicity of abnormal hematopoietic cells in the bone marrow. DNA hypomethylation of aberrantly methylated genes involved in normal cell cycle regulation, differentiation and death pathways may result in gene re-expression and restoration of cancer-suppressing functions to cancer cells. The cytotoxic effects of VIDAZA cause the death of rapidly dividing cells, including cancer cells that are no longer responsive to normal growth control mechanisms. Non-proliferating cells are relatively insensitive to VIDAZA

About Celgene

Celgene Corporation 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. For more information, please visit the company's website at www.celgene.com.

VIDAZA is a registered trademark of Celgene Corporation.

    
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    (1) Fenaux, P. et al. Efficacy of azacitidine compared with that of
        conventional care regimens in the treatment of higher-risk
        myelodysplastic syndromes: a randomized, open-label, phase III study.
        The Lancet Oncology. 2009. Volume 10, Issue 3, Pages 223-232.
    

For further information: FOR MEDIA INTERVIEWS, PLEASE CONTACT: Rosalind O'Connell, Hill and Knowlton Canada, [phone], [email protected]