SOUTH SAN FRANCISCO, Calif., Oct. 31, 2013 (GLOBE NEWSWIRE) -- Hyperion Therapeutics, Inc. (Nasdaq:HPTX) today announced that it has begun enrolling patients in its THRIVE study, a long term registry of patients with urea cycle disorders (UCDs) which will capture clinical outcomes and comparative effectiveness data.
The THRIVE study is a multi-center, prospective, non-interventional study designed to collect data in up to 500 UCD patients as part of their routine management. The study will track long-term outcomes, including hyperammonemic crises, ammonia levels, growth and development, and neurocognitive outcomes. Eligible patients will be enrolled in the study and followed for up to 10 years with annual interim data reports planned. At the time of enrollment, retrospective and baseline data will be collected. Data will also be collected during routine office visits. In addition, the study will capture the duration, dose, frequency, and route of administration of all current standard of care treatments, including ammonia-scavenging agents sodium phenylbutyrate, glycerol phenylbutyrate, or sodium benzoate. THRIVE offers the assistance of a Central Research Coordinating Office which facilitates participation and reduces the burden of study procedures on participating sites for all interested physicians and patients. Interested parties can visit the THRIVE website (www.thriveregistry.com) or contact thriveregistry@unitedbiosource.com to be included in the program.
"We are pleased to initiate this disease registry," said Bruce Scharschmidt, M.D., chief medical officer of Hyperion. "THRIVE meets one of our post marketing commitments to the FDA and will also contribute importantly to our understanding of UCDs and their management." "We have worked closely with the UCD community in designing this registry and are hopeful that it will help define best practices and potential improvements in management that will benefit patients both within and potentially outside the United States."
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 oral 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 unique from other ammonia removing medicines is that the active ingredient is released slowly in the body, mostly in the small intestine.
About BUPHENYL (sodium phenylbutyrate) Tablets and Powder
BUPHENYL is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). BUPHENYL should not be administered to patients with known hypersensitivity to sodium phenylbutyrate or any component of this preparation. The most common adverse reactions associated with BUPHENYL were amenorrhea dysfunction, decreased appetite, body odor (probably caused by its metabolite phenylacetate) and bad taste or taste aversion. Patients with urea cycle disorders should not take valproic acid, haloperidol, or steroids as these drugs have been reported to increase blood ammonia levels, and probenecid may affect the kidneys' excretion. Use with great care, if at all, in patients with congestive heart failure or severe renal insufficiency, and in clinical states where there is sodium retention with edema. Use caution when administering to patients with hepatic or renal insufficiency or inborn errors of beta oxidation. The safety or efficacy of doses in excess of 20 grams (40 tablets) per day has not been established.
Please see full Prescribing Information for BUPHENYL at http://ureacycle.com/buphenyl.aspx
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/files/RAVICTI_Prescribing_Information.pdf) and Medication Guide (http://www.ravicti.com/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 and hepatology. 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 in ex-U.S. geographies through business partners. 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, 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. Forward-looking statements contained in this press release include expectations about the THRIVE study. 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 Hyperion's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and any subsequent filings with the Securities and Exchange Commission.