June 25, 2014
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.
RAVICTI Safety Information
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 less than 2 months of age is contraindicated.
For additional Important Safety Information, including Warnings and Precautions, Adverse Events, Drug Interactions, and Special Populations, please see full Prescribing Information (http://www.ravicti.com/sites/default/files/RAVICTI_Prescribing_Information.pdf) and Medication Guide (http://www.ravicti.com/sites/default/files/RAVICTI_Medication_Guide.pdf ) for RAVICTI.
About Hyperion Therapeutics
Hyperion Therapeutics, Inc. is a commercial stage biopharmaceutical company committed to developing and delivering life-changing treatments for orphan diseases. The company’s first commercial product, RAVICTI® (glycerol phenylbutyrate) Oral Liquid, was approved in February 2013 and is currently being marketed in the United States. The company also owns worldwide rights to BUPHENYL® (sodium phenylbutyrate) Tablets and Powder, which it markets in the U.S. The compound is also marketed internationally through business partners. In addition, the company is developing glycerol phenylbutyrate for the potential treatment of hepatic encephalopathy and DiaPep277, a first-in-class immunotherapy for the potential treatment of new onset Type 1 diabetes. For more information, please visit www.hyperiontx.com.