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FDA panel backs Carbaglu

January 14, 2010 12:37 AM UTC

FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 12-0 on Wednesday that the risk-benefit profile of Carbaglu carglumic acid from Recordati S.p.A. (Milan:REC) supports its approval to treat hyperammonemia due to hepatic enzyme N-acetylglutamate synthetase (NAGS) deficiency, an inherited disorder that causes ammonia to accumulate in the blood. However, the panel was split 5-5, with two abstentions, on the issue of safety. According to an FDA spokesperson, the committee agreed that long-term safety studies are needed to fully understand the risks of Carbaglu and that the agency should address potential off-label use of the NAG synthetic analog. The panel also voted 11-1 and 12-0 that the data showed efficacy in acute and maintenance treatment, respectively. ...