About Hepatic Encephalopathy
HE is a serious but potentially reversible neurological disorder that can occur in patients with cirrhosis of any etiology or acute liver failure. HE comprises a spectrum of neurological signs and symptoms ranging from mild (e.g. minimal disorientation) to severe (e.g. coma, death) and is believed to occur when the brain is exposed to gut-derived toxins such as ammonia that are normally removed from the blood by a healthy liver. Based on the current epidemiological data, Hyperion estimates that approximately 140,000 cirrhosis patients have overt HE.
About Glycerol Phenylbutyrate
Glycerol phenylbutyrate, is a pre-pro-drug of phenylacetic acid, the active moiety of RAVICTI(R) and BUPHENYL(R), branded therapies currently FDA-approved as adjunctive therapy for the chronic management of patients with the most prevalent urea cycle disorders. Glycerol phenylbutyrate holds orphan product exclusivity in the United States and orphan designation in Europe for the maintenance treatment of patients with urea cycle disorders (UCD) and in the United States for the intermittent or chronic treatment of patients with cirrhosis and any grade of hepatic encephalopathy.
RAVICTI is indicated for use as a nitrogen binding agent for chronic management of adult and pediatric patients >=2 years of age with 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 (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia levels. The safety and efficacy of RAVICTI for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established. The use of RAVICTI in patients and Medication Guide ( ) for RAVICTI.
About Hyperion Therapeutics