BRISBANE, Calif., June 25, 2014 (GLOBE NEWSWIRE) -- Hyperion Therapeutics, Inc. (Nasdaq: HPTX ) 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/RA...mation.pdf ) and Medication Guide ( http://www.ravicti.com/sites/default/files/RA..._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 .
Forward-Looking Statements
To the extent that statements contained in this press release are not descriptions of historical facts regarding Hyperion, including statements related to the anticipated timing of receipt of an opinion from CHMP on our MAA, statements relating to the potential for, and timing of, approvals to market RAVICTI in Canada and the EU, statements relating to the potential therapeutic value of glycerol phenylbutyrate for the treatment of hepatic encephalopathy and statements related to the potential of DiaPep277 as a treatment for new onset Type 1 diabetes, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Hyperion undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties relating to the business of the company in general, see the "Risk Factors" section of Hyperion's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 7, 2014, and in any subsequent filings with the Securities and Exchange Commission.
Myesha Edwards Corporate Communications and Investor Relations (650) 745-7829
