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FDA Approves Azacitidine for the Treatment of Myelodysplastic Syndromes

ROCKVILLE, MD -- May 20, 2004 -- The Food and Drug Administration (FDA) today announced the approval of Vidaza (azacitidine) injection, the first effective treatment for patients with Myelodysplastic Syndrome (MDS). The product was given Fast Track Status and a priority review.

"By restoring normal growth and differentiation of bone marrow cells, this new treatment will offer a much needed option for patients suffering from this rare illness that, in some cases, has been found to progress to leukemia, a type of cancer. The agency continues to make approvals of these types of remarkable treatments one of its highest priorities," said Dr. Lester M. Crawford, Acting FDA Commissioner.

MDS is a collection of disorders in which the bone marrow does not function normally and not enough normal blood cells are made. MDS may develop following treatment with drugs or radiation therapy for other diseases, or it may develop without any known cause. Some forms of MDS can progress to acute myeloid leukemia (AML), a type of cancer in which too many white blood cells are made.

Vidaza is an orphan product. Orphan products are developed to treat rare diseases, or conditions that affect fewer than 200,000 people in the U.S. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first sponsor who obtains marketing approval for a designated orphan drug.

It is estimated that 7,000-12,000 new cases of MDS are diagnosed each year in the United States. Although it can occur in all age groups, the highest prevalence is in people over 60 years of age. Typical symptoms include weakness, fatigue, infections, easy bruising, bleeding and fever. MDS patients may need red blood cell and platelet transfusions, and antibiotic therapy for infections.

Vidaza is thought to work by restoring normal growth and differentiation of bone marrow cells.

The safety and effectiveness of Vidaza, in the treatment of all subtypes of MDS, were established in a randomized, controlled trial and in two non-randomized studies in a total of 268 patients. About 15% of patients in the three trials had complete or partial responses to azacitidine. Responses consisted of complete or partial normalization of blood counts and of immature cell percentages in the bone marrow. In responders the need for transfusions was eliminated.

The most common adverse events reported in clinical trials included nausea, anemia, thrombocytopenia (low platelets in blood), diarrhea, fatigue, irritation at the injection site, and constipation.

The sponsor and distributor of Vidaza is Pharmion Corporation, of Boulder, Colo.

Source: The Food and Drug Administration

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