The FDA ( U.S. Food and Drug Administration ) has approved Dacogen ( Decitabine ) injection for the treatment of myelodysplastic syndromes ( MDS ).
Dacogen is a new molecular entity that received orphan drug status.

The myelodysplastic syndromes 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 ).

An estimated 7,000 to 12,000 new cases of myelodysplastic syndromes 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, thrombocytopenia, anemia, fatigue, fever, nausea, cough, bleeding in the skin, constipation, diarrhea, and hyperglycemia.

Source: FDA, 2006


XagenaMedicine2006