Horizon Pharma plc, a biopharmaceutical company, 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 paediatric 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 paediatric 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 paediatric patients two months of age and older. Ravicti was approved by the US Food and Drug Administration in February 2013 for chronic management of UCDs in adult and paediatric patients two years of age and older.

Ravicti is not indicated for the treatment of acute hyperammonemia in patients with UCDs. The safety and efficacy of Ravictifor the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established. The use of Ravicti in patients < 2 months of age is contraindicated.