UPDATE: Hyperion Therapeutics Announces EMA Acceptance For Review of Marketing Authorization Application For RAVICTI

Hyperion Therapeutics, Inc.

today announced that the European Medicines Agency (EMA) has completed technical and content validation and accepted the filing of the Marketing Authorisation Application (MAA) for RAVICTI (glycerol phenylbutyrate) Oral Liquid for the treatment of urea cycle disorders (UCDs). Acceptance of the MAA confirms that the submission is complete so as to permit a substantive review by the Rapporteurs and the Committee for Medicinal Products for Human Use (CHMP). "We are very pleased to receive MAA acceptance for review of RAVICTI and anticipate a CHMP opinion in the third quarter of 2015," said Donald J. Santel, president and chief executive officer of Hyperion. "Collectively, our recent New Drug Submission acceptance by Health Canada and this MAA acceptance demonstrate significant progress toward our ongoing mission to make RAVICTI available to UCD patients outside of the United States." The MAA is supported by 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. 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 distinguishes RAVICTI from other currently available ammonia removing medicines is that it requires digestion such that the active ingredient enters the circulation more slowly.