Horizon Pharma Announces Availability Of RAVICTI (Glycerol Phenylbutyrate) Oral Liquid In Canada

DUBLIN, Ireland, Nov. 03, 2016 (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 and its affiliate Horizon Therapeutics Canada Limited, today announced that RAVICTI® (glycerol phenylbutyrate) Oral Liquid is now available in Canada.  In March 2016, Horizon received a Notice of Compliance (NOC) from Health Canada for RAVICTI 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).

“The availability of RAVICTI provides an important new treatment option for Canadians living with UCDs,” said Jared Rhines, vice president and general manager, Canada, LATAM and APAC, Horizon Pharma.  “Through our participation in UCD education and awareness programs around the world, our team has been inspired by the strength and resilience of people living with this rare and potentially devastating condition.  This interaction drives our commitment to support and partner with the UCD community, including the healthcare professionals, families and caregivers of people living with UCDs in Canada and beyond.”

UCDs 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.¹  UCD patients may experience episodes as a result of elevated ammonia levels in their blood – called hyperammonemic crises – which may result in irreversible brain damage, coma or death.^2,3  UCD symptoms may first occur at any age depending on the severity of the disorder, with more severe defects presenting earlier in life.⁴

“We have heard from so many patients and families about their challenges trying to manage this very rare and devastating condition.  We have heard over and over about the struggles to get a diagnosis and the feelings of isolation,” said Durhane Wong-Rieger, president and chief executive officer, Canadian Organization for Rare Disorders (CORD).  “Having new treatment options for those living with UCDs in Canada is important, and one of our top priorities at CORD is to help ensure that people living with rare diseases have access to innovative medicines.”

For more information about the availability of RAVICTI in Canada, including Horizon’s patient support programs, please call 1-844-823-4226.

About RAVICTI®

Indication and Clinical Use
In Canada, 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 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).

• Not for treatment of acute hyperammonemia in patients with UCDs.
• Safety and efficacy in patients with N-acetylglutamate synthase (NAGS) deficiency have not been established.
• Safety and efficacy in patients two months to two years of age have not been established.
• Clinical studies did not include sufficient subjects 65 years of age or older to determine response compared to younger subjects.
• RAVICTI should be prescribed by a physician experienced in the management of UCDs.

Contraindications

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

Most Serious Warnings and Precautions

• Neurologic Risk:  Acute Hyperammonemic Encephalopathy may occur even when patient is on therapy.  Symptoms of neurotoxicity are associated with the RAVICTI major metabolite, phenylacetic acid (PAA).

Other Relevant Warnings and Precautions

• Cardiovascular:  Caution should be observed in patients who have conditions that could be worsened by an increase in heart rate such as tachyarrhythmias or ischemic heart disease.
• Hepatic:  Caution should be used in patients with hepatic insufficiency.
• Pancreatic:  Caution should be used in patients in whom there is inadequate intestinal hydrolysis of RAVICTI as impaired absorption of PBA and hyperammonemia could occur.
• Renal:  Caution should be used in patients with renal insufficiency, including those with end-stage renal disease (ESRD) or those on hemodialysis.
• Pregnancy:  Caution should be used in patients who are pregnant as the potential risk to the fetus is not known.

For More Information:
Please consult the product monograph at http://webprod5.hc-sc.gc.ca/dpd-bdpp/index-eng.jsp for important information relating to adverse reactions, drug interactions, and dosing information which has not been discussed in this press release.

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.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the potential of RAVICTI to treat UCD patients.  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 whether RAVICTI will be successfully commercialized and sufficiently available in Canada, as well as those 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.

References:

1. Ah Mew, N, et al.  “Urea Cycle Disorders Overview.”  In: Pagon RA, et al., editors. GeneReviews®. Web. 31 Oct 2016.
2. Allen, Shari.  “Urea cycle disorder.”  Mental Health Clinician. 2.12. (2013): 398-401.  Web.  31 Oct 2016.
3. Summar, Ml, et al.  “Diagnosis, Symptoms, Frequency and Mortality of 260 Patients with Urea Cycle Disorders from a 21-Year, Multicentre Study of Acute Hyperammonaemic Episodes.”  Acta Paediatr. 97.10. (2008): 1420-1425. Web. 31 Oct 2016.
4. “Urea Cycle Disorders:  What Are Urea Cycle Disorders?”  NORD Physician Guides.  Web.  31 Oct 2016.

Contacts:

Investors:
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