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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