{Reference Type}: Journal Article
{Title}: Safety and efficacy of velaglucerase alfa replacement therapy for patients with type 1 Gaucher disease.
{Author}: Elstein D;Zimran A;
{Journal}: Expert Rev Endocrinol Metab
{Volume}: 8
{Issue}: 4
{Year}: Jul 2013
暂无{DOI}: 10.1586/17446651.2013.811871
{Abstract}: Gaucher disease is a multisystem disorder caused by deficiency of β-glucocerebrosidase. Exogenously delivered enzyme replacement therapy (ERT) is currently standard of care. Since 1994, intravenously delivered recombinant ERT with imiglucerase (Cerezyme; Genzyme Corporation, Cambridge, MA, USA) improves hematological, visceral and skeletal features of Gaucher disease at dosages of 15-60 units/kg bodyweight/infusion, administered every other week (EOW). Velaglucerase alfa (VPRIV®; Shire HGT, MA, USA) is a human wild-type-sequenced ERT produced in human cell lines using proprietary Gene-Activation® technology (Shire HGT). This article describes the results of a Phase I/II seminal trial in treatment-naive non-neuronopathic patients (including stepwise dose reduction to 30 units/kg/EOW) and three Phase III trials (two doses: 45 or 60 units/kg/EOW; switch-over from imiglucerase at identical dose; head-to-head with imiglucerase, 60 units/kg/EOW) and Phase III extension trial. Velaglucerase alfa was approved in 2010 in many countries; based on clinical trial experience, it is safe and effective in treatment-naive and switch-over patients, children and adults, splenectomized patients and those with an intact spleen.