Plant Cell Expressed Recombinant Glucocerebrosidase - taliglucerase alfa – as Therapy for Gaucher Disease in Patients Previously Treated with imiglucerase

Pastores, GM; Szer, Jeffrey; Petakov, Marijana; Cox, TM; Giraldo, Pilar; Rosenbaum, Hanna; Amato, Dominick; Mengel, Eugen; Chertkoff, Raul; Almon-Brill, Einat; Zimran, Ari

doi:10.1016/j.ymgme.2010.11.113

Cited by 1 publication

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“…Whether patients treated with imiglucerase would maintain stable disease parameters when switching to taliglucerase alfa was studied in a 9-month open-label switchover trial 8. During this trial, a severe worldwide shortage of imiglucerase (Cerezyme) occurred, which meant that many patients had their treatment with imiglucerase interrupted or were put on lower dosages 17.…”

Section: Clinical Trialsmentioning

confidence: 99%

“…Therefore the protocol was amended to allow assessment of stability based upon pre-shortage historical data and to treat a larger number of patients, including children. Preliminary results for 26 adult patients were reported at the WORLD conference 8. All patients received a dose of taliglucerase alfa that was identical to their previous imiglucerase dose, ranging from 10 to 60 U/kg every 2 weeks.…”

Section: Clinical Trialsmentioning

confidence: 99%

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An evidence-based review of the potential benefits of taliglucerase alfa in the treatment of patients with Gaucher disease

Hollak¹

2012

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Gaucher disease is an inherited lysosomal storage disorder, characterized by deficient activity of glucocerebrosidase leading to storage of glucocerebroside in tissue macrophages. Type I disease, the most prevalent form, lacks central nervous system involvement but presents primarily with variable degrees of hepatosplenomegaly, cytopenia, and bone disease. Intravenous enzyme replacement therapy can reverse these manifestations. In addition to the two enzymes currently authorized for use, the newest enzyme, taliglucerase alfa, is at the late stages of clinical development. Taliglucerase alfa is a unique product, as it is the first plant cell–based recombinant enzyme therapy. This review considers the existing evidence for therapeutic efficacy of taliglucerase alfa in the treatment of the non-neuronopathic manifestations of Gaucher disease. Clinical studies encompass one phase I trial in healthy volunteers, one phase III trial, and preliminary results from both an extension study and a switch study. In the 9-month, randomized, double-blind phase III trial, treatment-naïve patients with type I Gaucher disease were treated with either 30 or 60 U/kg every 2 weeks. Dose-dependent improvements were achieved after 6 and 9 months of therapy, with reductions in spleen and liver volumes and improvements in hemoglobin levels. Platelet counts improved initially only in the higher-dose group, but preliminary results from the extension study also show significant increases in the lower-dose group. Bone marrow involvement, as assessed by magnetic resonance imaging, improved in almost all patients. Taliglucerase alfa has shown a good safety profile, with few patients experiencing hypersensitivity reactions and developing antibodies. An additional enzyme replacement therapy for Gaucher disease would enable the treatment of more patients and would provide backup for unexpected production problems. Furthermore, it is expected that this new treatment would reduce the costs of therapy. Taliglucerase alfa is a valuable new treatment modality for the non-neuronopathic manifestations of Gaucher disease.

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Section: Clinical Trialsmentioning

confidence: 99%

Section: Clinical Trialsmentioning

confidence: 99%