New drug for bone marrow disease

FDA Consumer, July-August, 2004 by Zacitidine

The FDA has approved the first effective treatment for people with a collection of disorders called myelodysplastic syndrome (MDS), in which bone marrow does not function normally. Genetic abnormalities in people with the syndrome prevent bone marrow cells from maturing, and too many abnormal blood cells are made. Vidaza (azacitidine) injection, approved in May 2004, is thought to work by causing immature, abnormal bone marrow cells to become more mature, resembling normal cells.

The most common side effects of Vidaza reported in clinical trials include nausea, anemia, low platelets in the blood, diarrhea, fatigue, irritation at the injection site, and constipation.

MDS may develop without any known cause or following treatment with drugs or radiation therapy for other diseases. Some forms of MDS can progress to acute myeloid leukemia (AML), a type of cancer in which too many white blood cells are made.

It is estimated that 7,000 to 12,000 new cases of MDS are diagnosed each year in the United States. Though the disease can occur in all age groups, it occurs most often in people over 60. Typical symptoms include weakness, fatigue, infections, easy bruising, bleeding, and fever.

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