The Company Has Also Initiated a European Managed Access Program for RAVICTI
DUBLIN, IRELAND--(Marketwired - Jun 29, 2016) - Horizon Pharma plc (
RAVICTI is currently indicated for use as a nitrogen-binding agent for chronic management of adult and pediatric patients greater than two years of age with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. The sNDA seeks to expand the approved age range for RAVICTI to include children as young as two months of age, and is based on a clinical development program including two clinical trials that enrolled 17 UCD patients between the ages of two months and two years.
"This sNDA is an important milestone in the RAVICTI development program, and is consistent with our approach to reinvest in our medicines as we prioritize research to meet the unmet needs of people with devastating diseases," said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc. "Through our work with healthcare professionals and the UCD community, including patients and their families, we've learned that UCDs often strike early in a child's life and can be extremely challenging to manage, if not life threatening. In response, several studies were conducted evaluating the efficacy and safety of RAVICTI in very young children. We will now work with the FDA to determine whether the age range for RAVICTI can be expanded to include patients as young as two months of age."
In addition, Horizon recently partnered with Clinigen Group's Idis Managed Access (MA) division to initiate a Managed Access Program (MAP) in select European countries for eligible, individual patients living with UCDs. RAVICTI was approved in Europe in November 2015.
"Where local health authorities permit, the MAP will allow healthcare professionals access to RAVICTI while Horizon works through its country-by-country plan to make RAVICTI commercially available in Europe," continued Dr. Sherman.
Healthcare Professionals can obtain details about the RAVICTI Managed Access program by calling +44 (0) 1932 824 123, or emailing [email protected].
About Urea Cycle Disorders (UCDs)
UCDs are rare, 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.
About RAVICTI
In the U.S., RAVICTI was approved in February 2013 and is indicated for use as a nitrogen-binding agent for chronic management of adult and pediatric patients ≥ 2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI in the U.S. must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
Important Safety Information
LIMITATIONS OF USE
CONTRAINDICATIONS:
WARNINGS AND PRECAUTIONS:
ADVERSE REACTIONS:
DRUG INTERACTIONS:
Visit www.RAVICTI.com to download a copy of the RAVICTI Full Prescribing Information.
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, rheumatology and primary care business units. Horizon's global headquarters are in Dublin, Ireland. For more information, please visit www.horizonpharma.com. Follow @HZNPplc on Twitter or view careers on our LinkedIn page.
About Clinigen Group
Clinigen Clinical Trial Services is the global market leader in the management and supply of commercial medicines for clinical trials.
The Group is also the trusted global leader in ethically sourcing and supplying unlicensed medicines to hospital pharmacists and physicians for patients with a high unmet need, through three of its divisions: Idis Managed Access runs early access programs for innovative new medicines. Idis Global Access and Link Healthcare work directly with healthcare professionals to enable compliant access to unlicensed medicines on a global basis and niche essential licensed and generic medicines across Australasia, Africa and Asia (AAA region).
Clinigen Specialty Pharmaceuticals acquires global rights, revitalises and markets its own portfolio of niche hospital commercial products.
For more information, please visit www.clinigengroup.com
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding the potential of RAVICTI to treat UCD patients, the potential to expand RAVICTI's approved indication to include patients as young as two months of age, the expected impact of the MAP for RAVICTI access in Europe and Horizon Pharma's strategy and plans to commercialize RAVICTI in Europe. 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's sNDA will be approved, whether Horizon Pharma will be able to obtain adequate pricing and reimbursement and successfully commercialize RAVICTI in Europe or elsewhere outside of the United States, whether physicians outside of the United States will prescribe RAVICTI, whether RAVICTI will be sufficiently available in Europe through the MAP or otherwise, whether Horizon Pharma is able to maintain regulatory data and market protections and orphan exclusivity for RAVICTI in Europe, 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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