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U.S. Department of Health and Human Services

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FDA Approves Decitabine for Injection (Dacogen) to Treat Myelodysplastic Syndromes

On May 2, 2006, the U.S. Food and Drug Administration (FDA) approved decitabine for injection (Dacogen, MGI Pharma, Inc.) for the treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia) and intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

Safety and efficacy were demonstrated in an open-label, multi-center, randomized, controlled trial in 170 adult patients with all five French-American-British (FAB) subtypes of MDS and with International Prognostic Scoring System scores of High-Risk, Intermediate-2 and Intermediate-1. Eighty-nine patients were randomized to decitabine plus supportive care and 81 were randomized to supportive care. Patients randomized to the decitabine arm received the drug intravenously at a dose of 15 mg/m2 over a 3-hour period every 8 hours for 3 consecutive days. This treatment cycle was repeated every 6 weeks, depending on the patient’s clinical response and toxicity. Supportive care consisted of blood and blood product transfusions, prophylactic antibiotics, and hematopoietic growth factors.

Responses were classified using the MDS International Working Group criteria. Patients were required to be red blood cell and platelet transfusion independent during the period of response. The overall response rate (CR+PR) in the intent-to-treat population was 17% in the decitabine-treated group and 0% in the supportive care group (p<0.001). In the decitabine-treated group the median duration of response was 288 days and the median time to response was 93 days. All but one of the decitabine-treated patients who responded did so by the fourth cycle. Decitabine treatment did not significantly delay the median time to acute myelogenous leukemia or death.

A total of 164 patients were accrued to two additional open-label, single-arm, multi-center studies of decitabine in patients with any of the FAB subtypes of MDS. The overall response rates in these two studies were 26% (N=66) and 24% (N=98).

The major toxicity of decitabine was myelosuppression as manifested by neutropenia, thrombocytopenia, anemia, and febrile neutropenia. Other common adverse events included nausea, vomiting, diarrhea, constipation, fever, edema, hyperglycemia, hypomagnesemia, hypokalemia, arthralgias, back pain, cough, headache, insomnia, rash, petechiae, and pallor. No age- or gender- related differences in safety were detected. Patients with hepatic or renal dysfunction were not studied. Insufficient numbers of non-white patients were enrolled in these clinical trials to draw any conclusions.

Full prescribing information, including clinical trial information, safety, dosing, drug-drug interactions and contraindications is available at Drugs@FDA.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by phone at 1-800-FDA-1088; by facsimile 1-800-FDA-0178 by mail using the Form 3500