Introduction: Gaucher disease is the first lysosomal disease to benefit from enzyme replacement therapy, thus serving as model for numerous other lysosomal diseases. Alglucerase was the first glucocerebrosidase purified from placental extracts, and this was then replaced by imiglucerase - a Chinese hamster ovary cell-derived glucocerebrosidase.
Aim: The aim was to review the evidence underlying the use of imiglucerase in Gaucher disease type 1.
Evidence review: Data from clinical trials and Gaucher Registries were analyzed.
Conclusion: Imiglucerase has been prescribed and found to have an excellent efficacy and safety profile. We report herein the evidence-based data published for 26 years justifying the use of imiglucerase.
Keywords: Gaucher disease; imiglucerase; lysosomal disease; safety; therapeutic goals; treatment.