EU MA for treatment of Urea Cycle Disorders


EU Marketing Authorization for the treatment of Urea Cycle Disorders

Medical Need announced today that the European Commission on July 31, 2013, issued a marketing authorization in the EU for the product Pheburane for the treatment of Urea Cycle Disorders (UCD), involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase and argininosuccinate synthetase. Pheburane, a taste-masked coated formulation of sodium phenylbutyrate (NaPB), was developed by the French company Lucane Pharma, which will be the holder of the marketing authorization. Medical Need holds the exclusive distribution rights for the product in the Nordic, Baltic and Central Eastern European markets and will be responsible for the distribution, marketing and sale of the product in these territories.

Sodium phenylbutyrate is well established in the treatment of UCD. However, the currently available, uncoated formulation of the substance has an unpleasant bitter taste, which especially in children may compromise compliance or even make them refuse to take the treatment. Pheburane, with its taste-masked coated formulation, has been found to be well accepted by patients, including children, maintaining bioequivalence with the uncoated formulation.

“Pheburane addresses and resolves an important issue in the current treatment of UCD”, said Kennet Rooth, COO of Medical Need, and continued, “In addition to the improved palatability, which is a significant problem in particular for small children, the calibrated dosing spoon represents a practical tool for facilitating and optimizing the therapy for each individual patient. We are delighted to work with Lucane Pharma to improve the daily lives of the UCD patients and their families.”

Pierre Mambrini, CEO of Lucane Pharma, commented: “Lucane Pharma is delighted to entrust Pheburane to Medical Need, as the first collaboration in between our companies. Medical Need is a highly patient-oriented company, with an in depth knowledge in the metabolic field, and I am very optimistic about the success of this collaboration.”

About Urea Cycle Disorders (UCD)

The urea cycle is a vital metabolic process in the liver through which the body eliminates ammonium, which is formed when proteins are degraded. The process involves a series of enzyme reactions, resulting in the end product urea, which is secreted through the urine. In rare cases, children may inherit one or more deficient genes for a urea cycle enzyme from their parents. Malfunction in any of the enzymes leads to accumulation of excess ammonium in the body, which left untreated will cause irreversible brain damage, coma and ultimately death.

About Pheburane®

Pheburane is a newly developed taste-masked, coated, granular formulation of sodium phenylbutyrate (NaPB). NaPB acts by removing ammonium through an alternative pathway and is well-established in the treatment of UCD. The currently available uncoated formulation of NaPB has an unpleasant bitter taste which, especially in children, may compromise compliance or even lead to discontinuation and thereby impair the metabolic control. Pheburane is delivered with a calibrated dosing spoon to facilitate accurate and flexible dosing for patients and caregivers.