SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Hyperion Therapeutics, Inc. today announced that it has acquired worldwide rights to the investigational drug Ravicti™ (glycerol phenylbutyrate) from Ucyclyd Pharma, Inc., a wholly owned subsidiary of Medicis Pharmaceutical Corporation (NYSE: MRX). Terms of the deal were not disclosed. In connection with the acquisition announced today, Hyperion also entered into an amended and restated collaboration agreement with Ucyclyd pursuant to which Hyperion retains an option to acquire, in the first half of 2013 for a pre-negotiated price, worldwide rights for BUPHENYL® and, subject to certain conditions, AMMONUL®.
Under terms of a previous collaboration agreement with Ucyclyd Pharma, Hyperion has been developing Ravicti for two orphan diseases: urea cycle disorders and episodic hepatic encephalopathy. A New Drug Application (NDA) for the use of Ravicti as adjunctive therapy for the chronic management of urea cycle disorders in patients six years of age and older was recently accepted for filing by the FDA and is currently under review. The FDA action date under the Prescription Drug User Fee Act (PDUFA) is October 23, 2012. Hyperion has completed enrollment in a phase II study in patients with cirrhosis and episodic hepatic encephalopathy. Results from that study are expected to be available late in the second quarter of this year.
Ravicti™ (Glycerol Phenylbutyrate) UCD Development Program
The Ravicti NDA includes results from a single Phase III study which included a long term safety extension and two Phase II supporting studies. The Phase III multi-center, randomized, double-blind, placebo-controlled, cross-over study evaluated the non-inferiority of Ravicti as compared to sodium phenylbutyrate (BUPHENYL®) in controlling blood ammonia in adults aged 18 years and above with UCD. The study was conducted in accordance with a Special Protocol Assessment (SPA) with the FDA.
Ravicti™ (glycerol phenylbutyrate), an investigational drug formerly known as HPN-100, is a pre-pro-drug of phenylacetic acid, the active moiety of BUPHENYL®, the only branded therapy currently FDA-approved as adjunctive therapy for the chronic management of patients with urea cycle disorders due to deficiencies in carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), and argininosuccinic acid synthetase (AS). Ravicti holds orphan product designations in the US and Europe for the maintenance treatment of patients with urea cycle disorders and in the US for the intermittent or chronic treatment of patients with cirrhosis and any grade of hepatic encephalopathy.
About Urea Cycle Disorders
Urea cycle disorders are inherited, inborn errors of metabolism present in an estimated 1 in 10,000 births in the United States. Patients with urea cycle disorders are deficient in one of the key enzymes that comprise the urea cycle, the body’s primary vehicle for removing ammonia, a potent neurotoxin, from the bloodstream. Onset may occur at any age depending on the severity of the disorder. If left untreated, urea cycle disorders can cause dangerously heightened levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma, and/or death.
About Hepatic Encephalopathy
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 literature, Hyperion estimates that there are approximately one million1,2 patients in the US with cirrhosis, of whom approximately 140,000 have overt HE.
About Hyperion Therapeutics
Hyperion Therapeutics is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat disorders in the areas of orphan diseases and hepatology. Hyperion is developing Ravicti™ (glycerol phenylbutyrate) for two orphan indications: urea cycle disorders (UCD) and hepatic encephalopathy. Hyperion is headquartered in South San Francisco, CA.
BUPHENYL® is a registered trademark of Ucyclyd Pharma, Inc.
AMMONUL® is a registered trademark of Ucyclyd Pharma, Inc.
Ravicti™ is a trademark of Hyperion Therapeutics, Inc.
Full Prescribing Information for BUPHENYL® is available at www.Buphenyl.com or by contacting Ucyclyd Pharma, Inc.
Full Prescribing Information for AMMONUL® is available at www.Ammonul.com or by contacting Ucyclyd Pharma, Inc.
1 Bell BP, Manos MM, Zaman A, et al. The epidemiology of newly diagnosed chronic liver disease in gastroenterology practices in the United States: results from population-based surveillance. Am J Gastroenterol 2008; 103:2727-2735.
2 Dufour MC. Chronic liver disease and cirrhosis. In digestive diseases in the United States: epidemiology and impact. JE Everhart, Editor, 1994; NIH publication No. 94-1447:615-646.