The Medication(s) listed below have been approved by the Food & Drug Administration (FDA) as orphan products for treatment of this condition. There may be other products available
that are not considered orphan products.
Glycerol phenylbutyrate (Brand name: Ravicti)
- Manufactured by
Horizon Pharma, Inc.
FDA-approved indication: Use as a nitrogen-binding adjunctive therapy for chronic management of adult and pediatric patients at least 2 months of age with urea cycle disorders (UCDs) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (eg, essential amino acids, arginine, citrulline, protein-free calorie supplements).
National Library of Medicine Drug Information Portal