Velaglucerase alfa: a new option for Gaucher disease treatment.

Abstract

Type 1 Gaucher disease (GD) results from inherited β-glucocerebrosidase gene mutations, leading to anemia, thrombocytopenia, splenomegaly, hepatomegaly and skeletal disease. Velaglucerase alfa is a β-glucocerebrosidase produced by gene activation in a human cell line, and indicated for type 1 GD. A phase I/II clinical trial (TKT025; N = 12), its ongoing extension (TKT025EXT) and three phase III trials (total N = 82), showed that velaglucerase alfa is generally well tolerated in adult and pediatric patients. Many disease-related parameters improved significantly in two phase III trials in treatment-naïve patients, and were successfully maintained in imiglucerase-experienced patients in a phase II/III switch study. Ten adults in TKT025EXT sustained improvements through 5 years, including bone mineral density. Comparison with imiglucerase shows that velaglucerase alfa is an effective, generally well-tolerated alternative enzyme replacement therapy. In vitro data suggest velaglucerase alfa may be internalized into cells more efficiently and have a lower rate of seroconversion. However, these results do not necessarily correlate with clinical efficacy.

• Authors: A Zimran
• Journal: Drugs of today (Barcelona, Spain : 1998) (Drugs Today (Barc)) Vol. 47 Issue 7 Pg. 515-29 (Jul 2011) ISSN: 1699-3993 [Print] Spain
• PMID: 22013559 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright 2011 Prous Science, S.A.U. or its licensors. All rights reserved.

Chemical References

• Glucosylceramidase
• Velaglucerase alfa, human

Topics

• Animals
• Clinical Trials as Topic
• Gaucher Disease (drug therapy)
• Glucosylceramidase (adverse effects, pharmacokinetics, pharmacology, therapeutic use)
• Humans