An overview of RAVICTI® (glycerol phenylbutyrate) Oral Liquid

Review efficacy data, safety information, mechanism of action, and dosing and administration for RAVICTI


Effective ammonia control through life’s busy moments1

RAVICTI is the only FDA-approved oral therapy clinically studied over the long term (12 months) in pediatric and adult patients with urea cycle disorders (UCDs).1

References:  1. RAVICTI [package insert]. Lake Forest, IL: Horizon Pharma USA, Inc.; 2018.  2. Data on file. Horizon Pharma USA, Inc.; 2015.   3. Monteleone JPR, Mokhtarani M, Diaz GA, et al. Population pharmacokinetic modeling and dosing simulations of nitrogen-scavenging compounds: disposition of glycerol phenylbutyrate and sodium phenylbutyrate in adult and pediatric patients with urea cycle disorders. J Clin Pharmacol. 2013;53(7):699-710. doi:10.1002/jcph.92.   4. Lee B, Diaz GA, Rhead W, et al. Blood ammonia and glutamine as predictors of hyperammonemic crises in patients with urea cycle disorder. Genet Med. 2015;17(7):561-568. doi:10.1038/gim.2014.148.   5. Lichter-Konecki U, Diaz GA, Merritt JL II, et al. Ammonia control in children with urea cycle disorders (UCDs); phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate. Mol Genet Metab. 2011;103(4):323-329. doi:10.1016/j.ymgme.2011.04.013.  6. The Physician’s Guide to Urea Cycle Disorders. National Organization for Rare Disorders website. 2012. Accessed May 3, 2017.   7. Braissant O, McLin VA, Cudalbu C. Ammonia toxicity to the brain. J Inherit Metab Dis. 2013;36(4):595-612. doi:10.1007/s10545-012-9546-2.   8. Mokhtarani M, Diaz GA, Rhead W, et al. Elevated phenylacetic acid levels do not correlate with adverse events in patients with urea cycle disorders or hepatic encephalopathy and can be predicted based on the plasma PAA to PAGN ratio. Mol Genet Metab. 2012;107(3):308-314. doi:10.1016/j.ymgme.2012.08.006.   9. Mokhtarani M, Diaz GA, Rhead W, et al. Urinary phenylacetylglutamine as dosing biomarker for patients with urea cycle disorders. Mol Genet Metab. 2012;107(3):308-314. doi:10.1016/j.ymgme.2012.08.006.   10. Diaz GA, Krivitzky LS, Mokhtarani M, et al. Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate. Hepatology. 2013;57(6):2171-2179. doi:10.1002/hep.26058.

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