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A systematic review on effectiveness and safety of eliglustat for type 1 Gaucher disease

Introduction: Eliglustat is a novel therapeutic agent for the treatment of Gaucher type 1 disease (GD1). GD1 is the most prevalent form of Gaucher disease, resulting from deficient activity of glucocerebrosidase. This leads to lysosomal storage of glucosylceramide, causing hepatosplenomegaly, bone disease and cytopenia. Eliglustat exerts it beneficial effects by inhibiting glucosylceramide synthesis (substrate reduction therapy), but does not cross the blood–brain barrier. Areas covered: This systematic review describes the currently available literature and data in the public domain on the clinical effectiveness and toxicity of eliglustat for the treatment of GD1 as well as the safety and tolerability in healthy controls (18 November 2013). Expert opinion: Eliglustat shows high promise as an oral treatment for GD1, with robust effectiveness on all relevant disease parameters in clinical trials. Oral treatment may offer a convenient alternative to patients who are treated lifelong with enzyme replacement therapy (ERT). Currently, insufficient data are available to establish superiority of eliglustat over ERT, especially on long-term complications and effectiveness in GD1 patients with severe disease, active bone disease or polyneuropathy. Eliglustat does not cause gastrointestinal side effects as observed with miglustat. Frequently prescribed concomitant CYP2D6-inducing medication and cardiovascular disease may restrict prescription of eliglustat.