Yves Kibleur1, Nathalie Guffon2. 1. Lucane Pharma, 172 rue de Charonne, 75011, Paris, France. ykibleur@lucanepharma.com. 2. Hôpital Femme-Mère-Enfant, Centre de Référence des maladies héréditaires du métabolisme, 59 Boulevard Pinel, 69577, Bron Cedex, France.
Abstract
OBJECTIVES: The aim was to describe the status of patients with urea cycle disorders (UCD) at the latest long-term clinical follow-up of treatment with a new taste-masked formulation of sodium phenylbutyrate (NaPB) granules (Pheburane). These patients are a subset of those treated under a cohort temporary utilisation study (ATU) previously reported and now followed for 2 years. METHODS: From a French cohort temporary utilization authorization (ATU) set up to monitor the use of Pheburane on a named-patient basis in UCD patients in advance of its marketing authorization, a subset of patients were followed up in the long term. Data on demographics, dosing characteristics of NaPB, concomitant medications, adverse events and clinical outcomes were collected at a follow-up visit after 1-2 years of treatment with the drug administered under marketing conditions. This paper reports on the subset of patients who were included in further long-term follow-up at the principal recruiting metabolic reference center involved in the original cohort. RESULTS: No episode of metabolic decompensation was observed over a treatment period ranging from 8 to 30 months with Pheburane, and the range of ammonia and glutamine levels continued to improve and remained within the normal range, thus adding valuable longer-term feedback to the original ATU report. In all, no adverse events were reported with Pheburane treatment. These additional data demonstrate the maintenance of the safety and efficacy of Pheburane over time. CONCLUSIONS: The recently developed taste-masked formulation of NaPB granules (Pheburane) improved the quality of life for UCD patients. The present post-marketing report on the use of the product confirms the original observations of improved compliance, efficacy and safety with this taste-masked formulation of NaPB.
OBJECTIVES: The aim was to describe the status of patients with urea cycle disorders (UCD) at the latest long-term clinical follow-up of treatment with a new taste-masked formulation of sodium phenylbutyrate (NaPB) granules (Pheburane). These patients are a subset of those treated under a cohort temporary utilisation study (ATU) previously reported and now followed for 2 years. METHODS: From a French cohort temporary utilization authorization (ATU) set up to monitor the use of Pheburane on a named-patient basis in UCD patients in advance of its marketing authorization, a subset of patients were followed up in the long term. Data on demographics, dosing characteristics of NaPB, concomitant medications, adverse events and clinical outcomes were collected at a follow-up visit after 1-2 years of treatment with the drug administered under marketing conditions. This paper reports on the subset of patients who were included in further long-term follow-up at the principal recruiting metabolic reference center involved in the original cohort. RESULTS: No episode of metabolic decompensation was observed over a treatment period ranging from 8 to 30 months with Pheburane, and the range of ammonia and glutamine levels continued to improve and remained within the normal range, thus adding valuable longer-term feedback to the original ATU report. In all, no adverse events were reported with Pheburane treatment. These additional data demonstrate the maintenance of the safety and efficacy of Pheburane over time. CONCLUSIONS: The recently developed taste-masked formulation of NaPB granules (Pheburane) improved the quality of life for UCD patients. The present post-marketing report on the use of the product confirms the original observations of improved compliance, efficacy and safety with this taste-masked formulation of NaPB.
Authors: George A Diaz; Lauren S Krivitzky; Masoud Mokhtarani; William Rhead; James Bartley; Annette Feigenbaum; Nicola Longo; William Berquist; Susan A Berry; Renata Gallagher; Uta Lichter-Konecki; Dennis Bartholomew; Cary O Harding; Stephen Cederbaum; Shawn E McCandless; Wendy Smith; Gerald Vockley; Stephen A Bart; Mark S Korson; David Kronn; Roberto Zori; J Lawrence Merritt; Sandesh C S Nagamani; Joseph Mauney; Cynthia Lemons; Klara Dickinson; Tristen L Moors; Dion F Coakley; Bruce F Scharschmidt; Brendan Lee Journal: Hepatology Date: 2013-01-03 Impact factor: 17.425
Authors: Javier De Las Heras; Luis Aldámiz-Echevarría; María-Luz Martínez-Chantar; Teresa C Delgado Journal: Expert Opin Drug Metab Toxicol Date: 2016-11-28 Impact factor: 4.481