Genzyme Gaucher drug improves bone density

Cerezyme, an enzyme replacement therapy, is the standard of care for patients with type 1 Gaucher disease. Patients with Gaucher disease are at increased risk for developing bone complications.

The study compared the outcomes of patients with type 1 Gaucher disease who received treatment with Cerezyme to patients who did not receive treatment. Both the treated and untreated patients had bone mineral density values significantly below normal at baseline.

The analysis found that patients receiving Cerezyme experienced a significant increase in bone mineral density over time when compared with patients who were not treated. Furthermore, the improvements in bone mineral density were found to be dose-dependent, with those patients receiving higher doses of Cerezyme achieving greater increases in bone mineral density.

The investigators found that achieving normal bone mineral density scores may require eight years or more of sustained treatment with Cerezyme, longer than is required to reach normal values for other disease parameters, such as hematological parameters or reductions in liver and spleen volume. They concluded that a normal bone mineral density score should be a therapeutic goal for patients with type 1 Gaucher disease.