People with MDS have bone marrow that does not produce enough mature blood cells, resulting in a lack of healthy blood cells that can function properly in the body.
Dacogen is believed to work by promoting normal development of blood cells. It is a new molecular entity that received orphan drug status from the FDA.
Orphan products are developed to treat rare diseases or conditions that affect fewer than 200,000 people in the U.S.
Steven Galson, MD, Director of FDA's Center for Drug Evaluation and Research, says, "Today's approval of Dacogen offers patients with this rare disease an additional treatment option that may help these patients avoid blood transfusions."
MDS can develop following treatment with drugs or radiation therapy for other diseases or it can 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.
