The Food and Drug Administration (FDA) has expanded the approval of Ravicti (glycerol phenylbutyrate; Horizon Pharma) to include infants <2 months old. Ravicti, a nitrogen-binding agent, is indicated for the chronic management of urea cycle disorders (UCDs) in adults and children who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

The approval was based on a study that assessed the safety, efficacy, and pharmacokinetics of Ravicti in 16 patients with UCDs less than 2 months of age. Ten of the 16 patients transitioned from sodium phenylbutyrate to Ravicti, 3 were transitioned from intravenous sodium benzoate and sodium phenylacetate, and 3 were treatment-naïve. Of the 16 patients, 16, 14, 12, 6, and 3 patients were treated for 1, 3, 6, 12, and 18 months, respectively.

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Results showed patients treated with Ravicti maintained stable ammonia levels relative to their pre-study enrollment. In addition, mean ammonia levels were lower during treatment with Ravicti compared to baseline values. Adverse reactions reported in at least 10% of patients <2 months old were vomiting, rash, gastroesophageal reflux, increased hepatic enzymes, feeding disorder (decreased appetite, hypophagia), anemia, cough, dehydration, metabolic acidosis, thrombocytosis, thrombocytopenia, neutropenia, lymphocytosis, diarrhea, flatulence, constipation, pyrexia, lethargy, and irritability/agitation.

“The FDA approval of Ravicti for children younger than 2 months provides a new alternative for the management of patients with a UCD that is easy to dose and administer to infants given the liquid formulation,” said Nicola Longo, MD, PhD, clinical geneticist at Primary Children’s Hospital and the University of Utah Hospital, and a lead investigator of a clinical study evaluating Ravicti for newborns.

Ravicti is supplied as a 1.1g/mL oral liquid in multi-use 25mL bottles.

For more information visit horizonpharma.com.