FOR IMMEDIATE RELEASE
May 3, 2006
Laura Alvey, 301-827-6242
FDA Approves New Treatment for Myelodysplastic Syndromes (MDS)
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
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.