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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: glycerol phenylbutyrate
Trade Name: RAVICTI
Date Designated: 04/27/2009
Orphan Designation: Maintenance treatment of patients with deficiencies in enzymes of the urea cycle
Orphan Designation Status: Designated/Approved
Horizon Pharma USA, INc.
150 South Saunders Road
Suite 400
Lake Forest, Illinois 60045
United States

The sponsor address listed is the last reported by the sponsor to OOPD.

Marketing approved:

1 Generic Name: glycerol phenylbutyrate
Trade Name: RAVICTI
Marketing Approval Date: 02/01/2013
Approved Labeled Indication: Use as a nitrogen-binding agent for chronic management of adult and pediatric patients > or =2 years of age with urea cycle disorders (UCDs) who 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).
Exclusivity End Date: 02/01/2020 
Exclusivity Protected Indication* :  Use as a nitrogen-binding adjunctive therapy for chronic management of adult and pediatric patients at least 2 years 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).
2 Generic Name: glycerol phenylbutyrate
Trade Name: RAVICTI
Marketing Approval Date: 04/28/2017
Approved Labeled Indication: For use as a nitrogen-binding agent for chronic management of patients 2 months of age and older with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
Exclusivity End Date: 04/28/2024 
Exclusivity Protected Indication* :  For use as a nitrogen-binding agent for chronic management of pediatric patients > or =2 months and <2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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