Horizon Pharma plc Receives Health Canada Approval for RAVICTI(R) (glycerol phenylbutyrate) Oral Liquid for the Treatment of Urea Cycle Disorders in Patients Two Years of Age and Older - Seite 2

Additionally, Horizon will provide RAVICTI via compassionate access until the end of 2016 for those without access to private or public insurance. It is anticipated that most private drug plans will provide coverage as soon as RAVICTI is available in Canada. Due to the standard six to seven month health technology assessment review process, it is anticipated that public drug plan coverage may not be available until the end of 2016.

About RAVICTI®
RAVICTI is now indicated for use in Canada as a nitrogen-binding agent for chronic management of adult and pediatric patients two years of age and older with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI should be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline and protein-free calorie supplements). RAVICTI is indicated for use in all 28 Member States of the EU and 3 Member States of the EEA for chronic management of adult and pediatric patients two months of age and older. RAVICTI was approved by the U.S. Food and Drug Administration in February 2013 for chronic management of UCDs in adult and pediatric patients two years of age and older.

RAVICTI should be prescribed by a physician experienced in the management of urea cycle disorders (UCDs).

Important Safety Information

LIMITATIONS OF USE:

• RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs
• 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 < 2 months of age is contraindicated

CONTRAINDICATIONS:
RAVICTI is contraindicated in patients who are:

• Hypersensitive to RAVICTI or its metabolites (phenylbutyric acid [PBA], phenylacetic acid [PAA], and phenylacetylglutamine [PAGN])
• Less than two months of age
• Breastfeeding

ADVERSE REACTION OVERVIEW:

• The incidence of serious adverse events in long term clinical trials with RAVICTI was 26% and consisted primarily of hyperammonemia (18%).
• The most common adverse drug reactions among all patients taking RAVICTI in clinical trials include diarrhea, flatulence headache, decreased appetite, vomiting, nausea, fatigue and skin odor.
• Adverse drug reactions that resulted in clinical intervention in UCD patients who participated in clinical trials were mostly gastrointestinal reactions (flatulence, nausea, vomiting, abdominal distention) or neurological reactions (dysgeusia, lethargy, speech disorder, paresthesia, tremor).

Detailed recommendations for the use of RAVICTI will be described in the product monograph, which will be made available by Health Canada on its website.