The MDS Foundation Says VIDAZA Approval In Europe Could Provide Survival Benefits To Patients With Myelodysplastic Syndromes And Related Cancers

Main Category: Cancer / Oncology
Also Included In: Regulatory Affairs / Drug Approvals
Article Date: 08 Jan 2009 - 4:00 PDT

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The Myelodysplastic Syndromes (MDS) Foundation applauds the European Commission's approval of VIDAZA (azacitidine) as an important advance for MDS patients in Europe. Vidaza represents an entirely new approach to treating cancer- it works by epigenetics, that is it helps restore the normal function of genes that regulate cell growth and development. The results, according to the AZA-001 clinical trial, show that VIDAZA is the first and only drug to show a meaningful survival increase for patients with high-risk MDS, and it can significantly reduce their dependence on blood transfusions. This is the largest international Phase III controlled study ever conducted in higher-risk MDS.

"This is a milestone for patients with MDS because VIDAZA has been shown to significantly increase their two-year survival rate compared to conventional care regimens, while it also frees 45 percent of patients from blood transfusions which can cause significant side effects and a significant decrease in quality of life," said Kathy Heptinstall, Operating Director of the Myelodysplastic Syndromes Foundation. "The MDS Foundation is committed to making sure that patients in-need are aware of and have access to new treatments that can help them to live longer with a better quality of life. We will support efforts to ensure reimbursement so that MDS patients across Europe have access to VIDAZA and all new treatment advances in MDS as they become available."

VIDAZA was approved for patients with specific types of MDS who are not eligible for stem cell transplants, including intermediate 2 and high risk MDS, chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML). Without VIDAZA, patients with higher risk MDS have a median survival of only about six to 12 months and often rely on blood transfusions to manage their symptoms. Repeated transfusions can expose patients to risks from cross contamination and also lead to a toxic buildup called "iron overload" that has the potential to severely damage the heart, liver and pancreas.

VIDAZA works through a process called epigenetics that regulates cell growth and development. This involves chemical changes on the DNA of certain cells that turn off or "silence" genes without changing the DNA sequences themselves. VIDAZA blocks these chemical changes in cells involved with MDS, to reactivate the genes and restore normal cell growth.

VIDAZA has been available in the United States for the last four years. The European approval was based upon data from the AZA-001 trial, which found that VIDAZA resulted in a mean survival of 24.4 months compared to 15 months for patients who received CCR. The trial also showed that VIDAZA delays the progression of MDS to a more serious form of leukemia known as AML. Roughly 30 percent of patient diagnosed with MDS will progress to AML. Data at a recent international cancer meeting also demonstrated improving benefits from continued use of VIDAZA, with almost half of patients achieving an improved response when treatment was continued for an additional four cycles.

About MDS

MDS is a primary neoplasm of the bone marrow that is more prevalent than any of the leukemias. MDS affects the function of blood cells; red blood cells, white blood cells or platelets. It is not known exactly how many people have MDS, however, according to current estimates, there are between 15,000 and 20,000 people diagnosed each year in Europe.

About the MDS Foundation

The Myelodysplastic Syndromes Foundation, Inc. is a multi-disciplinary, international organization devoted to the prevention, treatment, and study of the myelodysplastic syndromes. The organization is based upon the premise that international cooperation will accelerate the process leading to the control and cure of these diseases.

MDS Foundation

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