Abstract
Background: Friedreich's ataxia (FA) is a degenerative disease with an autosomal recessive inheritance pattern that has been related to the accumulation of iron within the mitochondria and the
formation of free radicals. The most common clinical manifestations are neurological alterations, cardiomyopathy and diabetes mellitus. Virtually all patients present with ataxia in the four limbs and gait alterations after the age of 5, although in some cases these alterations cases may occur earlier. The main cardiac manifestation is arrhythmia and complications caused by cardiomyopathy, which are a frequent cause of death, often around the age of 30-40 years. There is no specific treatment to correct the defect causing AF. Idebenone (Sovrima®) is a benzoquinone with a structure similar to the coenzyme Q10. It is a powerful antioxidant that binds to oxygen of free radicals and enhances the flow of electrons in the electron transfer chain. Idebedone was designated as an orphan drug by the European Medicines Agency (EMA) in 2004 and received a negative opinion for
commercialization in 2008 by the Committee for Medicinal Products for Human Use (CHMP) of the EMA mainly due to the lack of evidence regarding its efficacy in AF. This decision was ratified a few months later.
Objectives: To analyze the scientific evidence available on the efficacy and safety of idebenone
compared with any alternative treatment, including placebo, in patients with AF.
Methodology: Systematic review of scientific evidence available until July 2011. The internal validity and grade of recommendation of the included clinical trials were assessed by a reviewer using
Scottish Intercollegiate Guidelines Network (SIGN) criteria. A qualitative synthesis of the scientific evidence was carried out.
Results: The main data on the efficacy and safety of idebenone in patients with AF come from 11
clinical trials (4 comparative trials and 7 non-comparative trials). The results described regarding the main efficacy variables (variables of cardiac and neurological function) are discordant among clinical trials. Regarding safety, treatment with idebenone is generally safe and well-tolerated. The most common adverse effects are gastrointestinal disturbances. Isolated cases of neutropenia, transaminitis and tachycardia have been reported.
Conclusions: The evidence available in the literature confirms the safety of idebenone, but cannot prove its efficacy in the treatment of AF. Therefore, its use would only be recommended within the
context of a clinical trial.
Keywords
Friedreich Ataxia; Treatment; Idebenone
Bibliographic citation
Gómez D, Paladio N. Idebenona (Sovrima®) per al tractament de l’atàxia de Friedreich. Barcelona: Agència d‟Informació, Avaluació i Qualitat en Salut; 2012.
Audience
Professionals
Use this identifier for quote and/or link this document
https://hdl.handle.net/11351/3038This item appears in following collections
The following license files are associated with this item: