Horizon Pharma plc
Jul 14, 2017

Horizon Pharma plc Announces Four Poster Presentations on PROCYSBI® (Cysteamine Bitartrate) Delayed-Release Capsules at Cystinosis Research Network 2017 Family Conference

-- In a New Study Comparing PROCYSBI and Immediate-Release Cysteamine, Those Treated with PROCYSBI Had a 26 Percent Reduction in Exhaled Dimethyl Sulphide, Which is Associated with Halitosis --

DUBLIN, Ireland, July 14, 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 results from a new study evaluating the effects of treatment with PROCYSBI® (cysteamine bitartrate) delayed-release capsules or immediate-release cysteamine bitartrate on the breath of people living with nephropathic cystinosis.  In the study, patients receiving PROCYSBI had a 26 percent reduction in exhaled dimethyl sulphide - a metabolite of cysteamine bitartrate associated with halitosis (i.e., bad breath) - compared to those receiving immediate-release cysteamine.  The study is one of four PROCYSBI poster presentations that can be viewed at the Cystinosis Research Network (CRN) 2017 Family Conference in Snowbird, Utah, on Saturday, July 15.

"Treatment with cysteamine bitartrate may cause an unpleasant odor, particularly affecting the patient's breath, and can impact their willingness to maintain continuous cystine-depleting therapy," said Larry Greenbaum, M.D., Ph.D., division director, Department of Pediatric Nephrology, Emory University School of Medicine, who served as principal investigator for the study.  "This study is important because people living with nephropathic cystinosis need to be treated with cystine-depleting therapy early and continuously to avoid the serious and potentially life-threatening impact of the disease on tissues and organs."

Summary of Study Results:

Most frequently reported adverse events seen in ≥5 percent of patients taking PROCYSBI were vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue and rash.

"We are very pleased to support the Cystinosis Research Network's Family Conference, as these events provide an understanding of the many different challenges faced by people living with nephropathic cystinosis," said Jeffrey W.  Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc.  "This halitosis substudy is part of our ongoing effort to analyze the effects of PROCYSBI, because there are many considerations - such as the effect of a medicine on body odor and breath - that are very important for people working to manage their condition while living as normal a life as possible."

In addition to the halitosis substudy (Quantification of Dimethyl Sulphide Associated with Cysteamine Bitartrate-Induced Halitosis Using Breath Analysis in Cystinosis Patients Treated with Delayed-Release and Immediate-Release Cysteamine Bitartrate), the following encore presentations, which were presented at previous medical meetings, will be available for viewing at CRN:

About Cystinosis
Cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.  Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.  It is estimated that only about 2,000 people worldwide are currently diagnosed with cystinosis.  Nephropathic or "classic infantile" cystinosis - the most common and most severe form of the disease - is typically diagnosed in infancy and requires lifelong therapy.1

In the United States, PROCYSBI® (cysteamine bitartrate) delayed-release capsules is a cystine depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients two years of age and older.

Important Safety Information




The most common adverse reactions (≥5%) in patients treated in clinical trials are vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash and headache.



Lactation:  Breastfeeding is not recommended while taking PROCYSBI.

Please see the Full Prescribing Information at www.PROCYSBI.com.

To report SUSPECTED ADVERSE REACTIONS, contact Horizon Pharma at 1-866-479-6742 (Option 1) or FDA at 1-800-FDA-1088 or  www.fda.gov/medwatch.

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 PROCYSBI to treat patients with nephropathic cystinosis and the side effects of PROCYSBI.  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 future PROCYSBI results will be in line with prior PROCYSBI results and whether patients are willing to use PROCYSBI to treat nephropathic cystinosis, as well as those factors described in Horizon Pharma'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 Pharma does not undertake any obligation to update or revise these statements, except as may be required by law.


  1. Cystinosis Research Foundation.  "About cystinosis."  Available at http://www.cystinosisresearch.org/About-Cystinosis/.  Accessed July 12, 2017.

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