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The Food and Drug Administration (FDA) today approved Dacogen (decitabine) injection for the treatment of myelodysplastic syndromes (MDS). Dacogen is a new molecular entity that received orphan drug status. 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.
Patients with MDS have bone marrow that does not produce enough mature blood cells. This causes a lack of healthy blood cells that can function properly in the body. Dacogen is thought to work by promoting normal development of blood cells. Different types of MDS exist, resulting in different manifestations of the disease. For example, some patients with MDS require chronic blood transfusions.
"Today's approval of Dacogen offers patients with this rare disease an additional treatment option that may help these patients avoid blood transfusions," said Steven Galson, MD, Director of FDA's Center for Drug Evaluation and Research.
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.
An estimated 7,000 to 12,000 new cases of MDS are diagnosed yearly in the United States. Although MDS occurs 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.
The safety and effectiveness of Dacogen were demonstrated in a randomized, controlled trial where patients received either Dacogen or the standard therapy and in two non-randomized studies where all of the patients received Dacogen. The new drug was evaluated in a total of 268 patients. About 22% of patients in the three trials had complete or partial responses to Dacogen. Responses consisted of complete or partial normalization of blood counts and of fewer immature cells in the bone marrow. In responders the need for transfusions was eliminated during the period of response.
The most common side effects reported in clinical trials included neutropenia (low white blood cell count), thrombocytopenia (low platelets in blood), anemia, fatigue, fever, nausea, cough, bleeding in the skin, constipation, diarrhea, and hyperglycemia (high blood sugar).
Dacogen is manufactured by Pharmachemie B.V. Haarlem, The Netherlands for MGI PHARMA, INC., Bloomington, MN.
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