FDA approved treatments for Type 1 Gaucher Disease revolve around two approaches.
Neither of these approaches addresses the fatal neurological deterioration that accompanies Type 2 and Type 3 disease. These patients have no therapeutic options
Enzyme Replacement Therapy (ERT) (intravenous): Biologics aimed to replace the critical enzyme deficiency; administered through IV infusion every 2 to 4 weeks
- 3 drugs: Marketed Sanofi (Genzyme), Pfizer, and Shire
- ~$1.5 billion in total annual sales
Substrate Reduction Therapy (SRT) (oral): Small molecule drugs aimed to decrease the accumulation of harmful storage material; administered through daily pills
- 2 drugs: Marketed by Sanofi (Genzyme) and Acetelion Pharma
- Significant side effects preventing adoption
- ~$150 million in total annual sales
- All 5 approved Type 1 drugs continue to show year over year growth
- The indication supports multiple drugs