Artigo Acesso aberto Revisado por pares

Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease

2011; Elsevier BV; Volume: 118; Issue: 22 Linguagem: Inglês

10.1182/blood-2011-07-366955

ISSN

1528-0020

Autores

Ari Zimran, Einat Brill‐Almon, Raul Chertkoff, Мilan Petakov, Francisco Blanco-Favéla, Eduardo Terreros Muñoz, Sergio Solorio, Dominick Amato, Gloria Durán, Fiorina Giona, René Heitner, Hanna Rosenbaum, Pilar Giraldo, Atul Mehta, Glen Park, Mici Phillips, Deborah Elstein, Gheona Altarescu, Mali Szleifer, Sharon Hashmueli, David Aviezer,

Tópico(s)

Glycosylation and Glycoproteins Research

Resumo

Abstract Taliglucerase alfa (Protalix Biotherapeutics, Carmiel, Israel) is a novel plant cell–derived recombinant human β-glucocerebrosidase for Gaucher disease. A phase 3, double-blind, randomized, parallel-group, comparison-dose (30 vs 60 U/kg body weight/infusion) multinational clinical trial was undertaken. Institutional review board approvals were received. A 9-month, 20-infusion trial used inclusion/exclusion criteria in treatment-naive adult patients with splenomegaly and thrombocytopenia. Safety end points were drug-related adverse events: Ab formation and hypersensitivity reactions. Primary efficacy end point was reduction in splenic volume measured by magnetic resonance imaging. Secondary end points were: changes in hemoglobin, hepatic volume, and platelet counts. Exploratory parameters included biomarkers and bone imaging. Twenty-nine patients (11 centers) completed the protocol. There were no serious adverse events; drug-related adverse events were mild/moderate and transient. Two patients (6%) developed non-neutralizing IgG Abs; 2 other patients (6%) developed hypersensitivity reactions. Statistically significant spleen reduction was achieved at 9 months: 26.9% (95% confidence interval [CI]: −31.9, −21.8) in the 30-unit dose group and 38.0% (95% CI: −43.4, −32.8) in the 60-unit dose group (both P < .0001); and in all secondary efficacy end point measures, except platelet counts at the lower dose. These results support safety and efficacy of taliglucerase alfa for Gaucher disease. This study was registered at www.clinicaltrials.gov as NCT00376168.

Referência(s)
Altmetric
PlumX