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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Urea cycle disorders


Other Names for this Disease

  • UCD
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Urea cycle disorders. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name Benzoate and phenylacetate
Trade Name
(Manufacturer Name)
Ammonul®
(Ucyclyd Pharma, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Adjunctive therapy in the treatment of acute hyperammonemia and associated encephalopathy in patients with deficiencies in enzymes of the urea cycle.
More Information about this product Drug Information Portal

Generic Name Sodium phenylbutyrate
Trade Name
(Manufacturer Name)
Buphenyl®
(Ucyclyd Pharma, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Adjunctive therapy in the chronic managment of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase.
More Information about this product Drug Information Portal

Generic Name Glycerol phenylbutyrate
Trade Name
(Manufacturer Name)
Ravicti
(Hyperion Therapeutics, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Use as a nitrogen-binding adjunctive therapy for chronic management of adult and pediatric patients > or = to 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).
More Information about this product Drug Information Portal

Generic Name Benzoate and phenylacetate
Trade Name
(Manufacturer Name)
Ucephan
(Immunex Immunex)
Indication
The FDA has approved this product to be used in this manner.
For adjunctive therapy in the prevention and treatment of hyperammonemia in patients with urea cycle enzymopathy due to carbamylphosphate synthetase, ornithine, transcarbamylase, or argininosuccinate synthetase deficiency.
More Information about this product Drug Information Portal

Other Names for this Disease
  • UCD
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.