A urea cycle disorder, or UCD, is a genetic disorder caused by a deficiency of one of the enzymes responsible for removing ammonia from the blood stream. If left untreated, high ammonia levels can lead to multiple organ failure and even prove fatal.
UCD is said to occur in approximately 1 in 10,000 births in the United States. Buphenyl Tablets and Powder, currently commercialized by Ucyclyd Pharma Inc., a wholly owned subsidiary of Medicis Pharmaceutical Corp. (MRX: Quote), is the only branded therapy approved by the FDA for chronic management of UCD.
At the altar, awaiting FDA decision is a new treatment for UCD - Ravicti, developed by Hyperion Therapeutics Inc. (HPTX: Quote), which is designed to address some of the limitations of Buphenyl.
For readers who are new to Hyperion, here's a brief overview of the upcoming event...
The New Drug Application for Ravicti for the chronic management of UCD in patients aged 6 years and above was submitted to the FDA last December based on data from a pivotal phase III trial in adult patients and the results of two phase II trials, one in adults and one in pediatric patients aged 6 through 17 years. As of now, the regulatory agency's decision date is set for October 23, 2012, unless it is extended.
The company submitted data from the switchover portion of a clinical trial in UCD patients aged 29 days through 5 years and a revised draft package insert requesting approval of Ravicti to include that patient population in April of this year. According to Hyperion, if the April 2012 update is classified as a major amendment by the FDA, the decision date may likely be delayed.
Hyperion acquired worldwide rights to Ravicti from Ucyclyd Pharma for undisclosed terms in March of 2012. The company also has an option to acquire, in the first half of 2013, worldwide rights for Buphenyl and, subject to certain conditions, Ammonul injection, the only adjunctive therapy currently FDA-approved for the treatment of acute hyperammonemia and associated encephalopathy in patients with urea cycle disorders, for an upfront payment of $22.0 million, plus subsequent milestone and royalty payments.
Ravicti is also being evaluated as a potential treatment for hepatic encephalopathy, or HE, a complex neuropsychiatric disorder that is caused by liver damage due to cirrhosis or hepatitis, which is also characterized by high blood ammonia. It is estimated that there are about 1 million patients in the U.S. with cirrhosis, of whom approximately 140,000 have overt HE. Salix Pharmaceuticals Inc.'s (SLXP: Quote) Rifaximin is the only therapy approved by the FDA to reduce recurrence of hepatic encephalopathy within the last 30 years.
Hyperion's hepatic encephalopathy clinical program comprises of two trials which have enrolled patients with cirrhosis. Last month, the company reported positive results from its phase II trial of Ravicti for HE. The company is planning to request an end of phase II meeting with the FDA in the fourth quarter of 2012, and receive guidance for a phase III trial in HE.
Ravicti has orphan drug designation for the indications of urea cycle disorder and hepatic encephalopathy.
A quick look at the company's balance sheet...
Since inception in November, 2006, the company has incurred losses, and as of March 31, 2012, had an accumulated deficit of $118.6 million. The company has not reported any revenue in the last three years. According to a recent SEC filing, revenues generated by the company in the years 2007 and 2008 were from commissions for promotion services through the Ucyclyd collaboration agreement related to the sales of Buphenyl and Ammonul for urea cycle disorder.
Hyperion listed its shares on the Nasdaq, going public as recently as July 26, offering its shares at $10 each. The stock has thus far hit a low of $9.95 and a high of $11.
Will Hyperion's lead drug candidate Ravicti clear the regulatory muster and reach pharmacy shelves? Stay tuned...
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by RTT Staff Writer
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