BRISBANE, Calif., June 3, 2014 (GLOBE NEWSWIRE) -- Hyperion Therapeutics, Inc. (Nasdaq:HPTX) today announced that the validation period is complete for its New Drug Submission (NDS) to Health Canada for RAVICTI (glycerol phenylbutyrate) Oral Liquid for the treatment of urea cycle disorders (UCDs), and that the RAVICTI NDS was granted Priority Review. Standard review in Canada takes 18 months or more and Priority Review, which is granted to promising medicines that address life-threatening or severely debilitating conditions, shortens the review time to approximately six months.
"We are pleased with the acceptance of the NDS with Priority Review for RAVICTI and potentially expanding the availability of RAVICTI to UCD patients outside of the United States," said Donald J. Santel, president and chief executive officer of Hyperion. "If approved, RAVICTI will be the first treatment approved by Health Canada to treat UCDs."
The NDS is based on results from 10 controlled and uncontrolled clinical trials studying the safety and efficacy of RAVICTI in 359 patients across three populations, including 114 UCD patients. Data from multiple Canadian patients enrolled at the University of Toronto were included in the submission. The safety and efficacy of RAVICTI is under review by Health Canada and market authorization has not yet been granted.UCD patients lack enzymes or transporters necessary for the conversion of ammonia to urea and experience heightened levels of ammonia in the bloodstream. Left untreated, UCDs can result in neurological damage, coma, and/or death. Approved by the Food and Drug Administration on February 1, 2013, RAVICTI is an odorless nearly tasteless liquid medication used for the long-term management of high blood levels of ammonia. In short term clinical studies involving more than 80 UCD patients ages two years and older, RAVICTI was safe and efficacious based on 24-hour ammonia profiles. In addition in long-term follow up studies, patients on RAVICTI maintained average fasting ammonia levels below the upper limit of normal. What makes RAVICTI different from other currently available ammonia removing medicines is that it requires digestion such that the active ingredient enters the circulation more slowly.