Zavesca Overview
Type 1 Gaucher Disease
Patients
Prescribing Information
Important Safety Information
Contact Us
ERT-Naive Patient Study Design* Extension Phase Design
  • 12-month open-label uncontrolled study
  • 28 adults with type 1 Gaucher disease who were unable or unwilling to take ERT and who had not taken ERT in the preceding 6 months
  • 22 patients completed the 12-month study
  • Zavesca 100 mg tid
  • 12-month open-label uncontrolled extension of ERT-Naive Patient Study
  • 18 patients who elected to continue on Zavesca 100 mg tid
  • 13 patients completed the extension phase

 

Zavesca Significantly Reduced Organ Volume
  • Significant reductions seen as early as month 61,2
  • Significant reductions over 24 months1,2

  Mean baseline value3:
2.38 L (n=27; 1 unknown)
Baseline range: 1.1 to 2.7 x normal

 

  Mean baseline value3:
1.66 L (n=20; 7 splenectomies; 1 unknown)
Baseline range: 5.1 to 24.8 x normal

Zavesca Produced Positive Hematological Changes
  • Improvements seen as early as month 61,2
  • Significant improvements seen at 24 months1,2
 

  Mean baseline value3:
12.28 g/dL (n=28; 9 [32%] anemic at baseline)
Anemia defined as hemoglobin concentration <11.5 g/dL

  Mean baseline value3:
88.10 x 109/L (n=28; 26 [93%] thrombocytopenic at baseline)
Thrombocytopenia defined as platelet
count <150 x 109/L

*This study is referred to as “Study 1” in the Zavesca package insert.
†All p values are changes from baseline.


1. Cox T, Lachmann R, Hollak C, et al. Novel oral treatment of Gaucher’s disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet. 2000;355:1481–1485.
2. Zavesca® (miglustat) package insert. Actelion Ltd. 2003.
3. Data on file, Actelion Pharmaceuticals US, Inc.

 
 



Full Prescribing Information | Important Safety Information | Contact Us