Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Clinigen Group's Idis Managed Access (MA) division and Horizon Pharma have initiated a Managed Access program in Europe for Horizon’s RAVICTI (glycerol phenylbuterate) Oral Liquid.
Subscribe to our email newsletter
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."