CHMP recommends approval of sodium phenylbutyrate (Pheburane®) for urea cycle disorders

Source: European Medicines Agency Area: News The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the granting of a marketing authorisation for sodium phenylbutyrate (Pheburane®) as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase or argininosuccinate synthetase.  It is indicated in all patients with neonatal-onset presentation, and in patients with late-onset disease who have a history of hyperammonaemic encephalopathy.   Sodium phenylbutyrate is converted in the body into phenylacetate, which combines with glutamine to form a substance that can be removed from the body by the kidneys. This results in reduced levels of nitrogen in the body. Pheburane® is a hybrid of Ammonaps®, which has been authorised in the EU since December 1999.
Source: NeLM - News - Category: Drugs & Pharmacology Source Type: news