SOURCE: Horizon Pharma plc

Horizon Pharma plc

March 21, 2016 07:00 ET

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

DUBLIN, IRELAND--(Marketwired - Mar 21, 2016) - 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 that Health Canada has issued a Notice of Compliance (NOC) for RAVICTI® (glycerol phenylbutyrate) Oral Liquid for use as an adjunctive therapy for chronic management of adult and pediatric patients two years of age and older with Urea Cycle Disorders (UCDs).

Last month Horizon announced that the Therapeutic Products Directorate of Health Canada had determined that RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid is eligible for data protection as it is an "innovative drug" as defined by section C.08.004.1 of the Food and Drug Regulations. Based on the approval, RAVICTI will be added to the Register of Innovative Drugs and receive data protection for a term of up to eight years from the date of the issuance of the NOC.

UCDs, which impact approximately 200 people in Canada, are inherited metabolic diseases caused by a deficiency of one of the enzymes or transporters that constitute the urea cycle. The urea cycle involves a series of biochemical steps in which ammonia, a potent neurotoxin, is converted to urea, which is excreted in the urine. UCD patients may experience episodes where they get symptoms from the ammonia in their blood being excessively high -- called hyperammonemic crises -- which may result in 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.

"The approval of RAVICTI in Canada is a significant milestone for Horizon Pharma as it continues to expand the accessibility of important medicines to patients with unmet treatment needs," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "I commend Health Canada for acknowledging the importance of this innovative medicine and the company intends to rapidly pursue funding and reimbursement. Horizon will soon be filing clinical and cost effectiveness submissions to the respective health technology assessment agencies in Canada and engaging with public and private drug plans to ensure that patients with Urea Cycle Disorders who may benefit from treatment are able to access RAVICTI as quickly as possible."

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.

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


  • 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

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


  • 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.

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 nine medicines through its orphan, primary care and specialty business units. Horizon's global headquarters are in Dublin, Ireland. For more information, please visit Follow @HZNPplc on Twitter or view careers on our LinkedIn page.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the potential of RAVICTI to treat UCD patients, the periods of regulatory data and market protection attaching to the approval for RAVICTI in Canada, potential reimbursement and availability of RAVICTI in Canada and Horizon Pharma's strategy and plans to commercialize RAVICTI in Canada. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include risks regarding whether Horizon Pharma will be able to obtain adequate pricing and reimbursement and successfully commercialize RAVICTI in Canada or elsewhere outside of the United States, whether physicians outside of the United States will prescribe RAVICTI, the availability and acceptance of competing products, whether Horizon Pharma is able to maintain regulatory data and market protections for RAVICTI in Canada, whether Horizon Pharma will be able to successfully execute its growth strategy outside of the United States, and other factors described in Horizon's filings with the United States Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in those filings. Forward-looking statements speak only as of the date of this press release and Horizon does not undertake any obligation to update or revise these statements, except as may be required by law.

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