FDA Drug Approval Summary: Azacitidine (5-azacytidine, Vidaza™) for Injectable Suspension

On May 19, 2004, azacitidine (5-azacytidine; VidazaTM; Pharmion Corporation, Boulder, CO, http://www. pharmion.com) for injectable suspension received regular approval by the U.S. Food and Drug Administration (FDA) for the treatment of all subtypes of myelodysplastic syndrome (MDS). This report summarizes the basis for this approval. Effectiveness was demonstrated in one randomized, controlled trial comparing azacitidine administered s.c. with best supportive care (observation group) and in two single-arm studies, one in which azacitidine was administered s.c. and in the other in which it was administered i.v. The dose of azacitidine, 75 mg/m2/day for 7 days every 28 days, was the same in all three studies. In the randomized trial, study participants were well matched with respect to age, sex, race, performance status, MDS subtype, and use of transfusion during the 3 months before study entry. Patients in the observation arm were permitted by protocol to cross over to azacitidine treatment if their disease progressed according to prespecified criteria. During the course of the study, more than half of the patients in the observation arm did cross over to the azacitidine treatment arm. The primary efficacy end point was the overall response rate. Response consisted of complete or partial normalization of blood cell counts and of bone marrow morphology. The response rate in the azacitidine arm was about 16%; there were no responses in the observation arm. The response rates in the two single-arm studies were similar (13% and 19%). The responses were sustained, with median durations of 11 months The Oncologist 2005;10:176–182 www.TheOncologist.com Correspondence: Edvardas Kaminskas, M.D., U.S. Food and Drug Administration, 5600 Fishers Lane, HFD-150, Rockville, Maryland 20857, USA. Telephone: 301-594-5753; Fax: 301-594-0499; e-mail: kaminskase@cder.fda.gov Received July 26, 2004; accepted for publication November 18, 2004. ©AlphaMed Press 1083-7159/2005/$12.00/0 The Oncologist FDA Commentary