Horizon Pharma plc
Mar 22, 2017

Horizon Pharma plc to Present Data on RAVICTI® (glycerol phenylbutyrate) Oral Liquid for Urea Cycle Disorder Patients Aged Two Months to Two Years

DUBLIN, Ireland, March 22, 2017 (GLOBE NEWSWIRE) -- Horizon Pharma plc (NASDAQ:HZNP), a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced the presentation of new data on the use of RAVICTI® (glycerol phenylbutyrate) Oral Liquid in children living with urea cycle disorders (UCD) two months to two years of age.  These data will be presented on Thursday, March 23 at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Phoenix, Arizona.

The study results are from two clinical trials that enrolled children with UCDs aged two months to less than two years.  Study participants were predominantly on relatively stable doses of sodium phenylbutyrate and switched to equivalent doses of RAVICTI.  The primary outcomes measured in the study were targeted mean normalized plasma ammonia of less than 100 μmol/L, hyperammonemic crises (HACs) and adverse events (AEs).  Safety and efficacy was also evaluated after patients transitioned to RAVICTI.

"The RAVICTI development program is part of our overall approach to reinvest in our medicines for people living with rare diseases," said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc.  "While UCDs are very rare and can be devastating, continued advancements in diagnosis and ongoing studies evaluating management options are helping improve the long-term outcome of people living with the disorder."

RAVICTI is currently indicated for use as a nitrogen-binding agent for chronic management of adult and pediatric patients two years of age or greater with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.  RAVICTI is not indicated for patients less than two years of age.  In June 2016, Horizon submitted a supplemental New Drug Applications (sNDA) with the U.S. Food and Drug Administration (FDA) seeking to expand the approved age range for RAVICTI to include children as young as two months of age.

The poster presentation (abstract #2873; poster #71), entitled "Safety and Efficacy of Glycerol Phenylbutyrate for Management of Urea Cycle Disorders in Patients Aged 2 Months to 2 Years," will be presented on Thursday, March 23, 2017 from 10:00 a.m. - 11:30 a.m. at the ACMG Annual Meeting.

About Urea Cycle Disorders
A UCD is a rare genetic disorder that affects approximately 1 in 35,000 live births in the United States.  It is caused by an enzyme deficiency in the urea cycle, a process that is responsible for converting excess ammonia from the bloodstream and ultimately removing it from the body.  Because of this, people with a UCD experience hyperammonemia, or elevated ammonia levels in their blood that can then reach the brain where it can cause irreversible brain damage, coma or death.  UCD symptoms may first occur at any age depending on the severity of the disorder, with more severe defects presenting earlier in life.1

About RAVICTI
In the U.S., RAVICTI was approved in February 2013 and is indicated for the 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).

Important Safety Information

LIMITATIONS OF USE

CONTRAINDICATIONS:
RAVICTI is contraindicated in patients:

WARNINGS AND PRECAUTIONS

USE IN SPECIFIC POPULATIONS

ADVERSE REACTIONS                 

DRUG INTERACTIONS

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit  www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see the Full Prescribing Information and Medication Guide for RAVICTI.

About Horizon Pharma plc
Horizon Pharma plc is a biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs.  The Company markets 11 medicines through its orphan, rheumatology and primary care business units.  For more information, please visit www.horizonpharma.com.  Follow @HZNPplc on Twitter or view careers on our LinkedIn page.

References:

  1. Ah Mew N, Lanpher BC, Gropman A, et al.; Urea Cycle Disorders Consortium. Urea Cycle Disorders Overview. 2003 Apr 29 [Updated 2015 Apr 9]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2017. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1217/
Contacts:

Tina Ventura

Senior Vice President, Investor Relations

Investor-relations@horizonpharma.com



U.S. Media Contact:

Matt Flesch

Executive Director, Product Communications

media@horizonpharma.com



Ireland Media Contact:

Ray Gordon

Gordon MRM

ray@gordonmrm.ie

Source: Horizon Pharma plc

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