A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase
Gregory M Pastores, Milan Petakov, Pilar Giraldo, Hanna Rosenbaum, Jeffrey Szer, Patrick B Deegan, Dominick J Amato, Eugen Mengel, Ee Shien Tan, Raul Chertkoff, Einat Brill-Almon, Ari Zimran
Blood Cells Molecules and Diseases | ACADEMIC PRESS INC ELSEVIER SCIENCE | Published : 2014
Taliglucerase alfa is a β-glucosidase enzyme replacement therapy (ERT) approved in the US and other countries for the treatment of Gaucher disease (GD) in adults and is approved in pediatric and adult patients in Australia and Canada. It is the first approved plant cell-expressed recombinant human protein. A Phase 3, multicenter, open-label, 9-month study assessed safety and efficacy of switching to taliglucerase alfa in adult and pediatric patients with GD treated with imiglucerase for at least the previous 2years. Patients with stable disease were offered taliglucerase alfa treatment using the same dose (9-60U/kg body weight) and regimen of administration (every 2weeks) as imiglucerase. Th..View full abstract
This study was sponsored by Protalix BioTherapeutics. Editorial and medical writing support was provided by Callie Grimes, PhD, of Peloton Advantage, LLC, and was funded by Pfizer. Pfizer and Protalix entered into an agreement in November 2009 to develop and commercialize taliglucerase alfa.