OREANDA-NEWS. Clinigen Group plc's (AIM: CLIN, 'Clinigen' or the 'Group') Idis Managed Access (MA) division and Horizon Pharma plc (NASDAQ: HZNP), have initiated a Managed Access program in Europe for Horizon's RAVICTI® (glycerol phenylbuterate) Oral Liquid.

RAVICTI received centralised marketing approval from the European Medicines Agency (EMA) in September 2015. It is indicated for the treatment of Urea Cycle Disorders (UCDs) in adults and children from two months old and over who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

The Managed Access program, run by Idis MA, will make RAVICTI immediately available to eligible patients living with UCDs in selected European countries prior to commercial launch.

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.

Steve Glass, Group Managing Director of Clinigen said:

"Urea cycle disorders can be serious and even life threatening so it is important that treatment options like RAVICTI can be accessed by those patients who need it.

"As the global leader in Managed Access programs, this partnership with Horizon enables us to use our expertise to support both Horizon and the UCD community in Europe, providing access to RAVICTI to help eligible patients who have no alternative option, ahead of the medicine's approval and commercial launch."

Jeffrey W. Sherman, M.D., FACP, Executive Vice President, Research and Development and Chief Medical Officer of Horizon Pharma plc said:

"As part of our efforts to address the unmet needs of people living with rare and challenging disorders, including UCDs, we're pleased to partner with Idis MA to make this Managed Access program available.

"Where local health authorities permit, the Managed Access program will allow healthcare professionals to have access to RAVICTI while Horizon works through its country-by-country plan to make RAVICTI commercially available in Europe."

About RAVICTI

In Europe, RAVICTI is now indicated for use in all 28 Member States of the EU and 3 Member States of the EEA as a nitrogen-binding agent for chronic management of adult and pediatric patients two months of age and older with UCDs who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.  

RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

About Clinigen Group

Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and services company with a unique combination of businesses focused on providing access to medicines. Its mission is to deliver the right medicine to the right patient at the right time. 

The Group consists of five synergistic businesses focused in three areas of global medicine supply; clinical trial, unlicensed and licensed medicines. 

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.


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.