Horizon Pharma plc Announces Urea Cycle Disorders Medical Foods and Supplements Financial Assistance Program

DUBLIN, Ireland, March 02, 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 support of a program developed by the National Organization for Rare Disorders (NORD) to help people with Urea Cycle Disorders (UCDs) seeking assistance with high out-of-pocket costs associated with the purchase of medical foods and supplements for their low-protein dietary needs.  

“Patients' health care needs extend beyond medications and we commend Horizon for being willing to support programs that are aimed at overcoming obstacles to other important treatments,” said Catherine Blansfield, RN, vice president of patient services at NORD.  “This donation provides financial support for products, which are essential for good health but, unfortunately, are not commonly covered by health insurance benefits.”

The program is independently administered by NORD and available to all people living with UCDs in the United States who meet the NORD eligibility criteria.  Horizon provided a charitable grant to help fund the program and has no involvement or input on its administration.

“I have to significantly limit the amount of protein I consume to avoid elevated ammonia levels, but the flipside is that too little protein can deprive my body of core amino acids, and that can also lead to high ammonia levels,” said Denise Z., who lives with a type of UCD called Citrullinemia Type 1.  “Medical foods and supplements that provide these core nutrients are vital to my health, and that is why this program is so important to those of us in the UCD community who struggle with the day-to-day challenges of living with a UCD.”

UCDs are rare genetic disorders that affect approximately 1 in 35,000 people in the United States.  It is caused by an enzyme deficiency in the urea cycle, a process that is responsible for converting excess ammonia from the bloodstream and ultimately removing it from the body.  Because of this, people with a UCD experience hyperammonemia, or elevated ammonia levels in their blood that can then reach the brain where it can cause 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.  Those with UCDs adhere to a low-protein diet to decrease the nitrogen load of the urea cycle, and receive essential nutrient supplementation to help achieve normal growth and metabolic stability.1

“At Horizon, we are always looking for ways to be supportive, beyond our therapies, for people with rare diseases,” said Robert Metz, senior vice president, business operations and external affairs, Horizon Pharma plc.  “We’ve received feedback directly from people living with UCDs, their caregivers and health care professionals about the financial burden that those who require medical foods and supplements face, and are thrilled to support NORD’s UCDs Medical Foods and Supplements Financial Assistance Program.”

To learn more about the UCDs Medical Foods and Supplements Financial Assistance Program, patients or their caregivers should contact NORD at 877-333-1860 or UCD@rarediseases.org.

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.

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Source: Horizon Pharma plc


  1. Ah Mew N, Lanpher BC, Gropman A, et al.; Urea Cycle Disorders Consortium. Urea Cycle Disorders Overview. 2003 Apr 29 [Updated 2015 Apr 9]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2017. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1217/