Initial $325 million Type 3 Gaucher Disease market opportunity without competition
Demonstrated ability to address an UNMET CLINICAL NEED In Type 3 Gaucher Disease patients
Type 3 Gaucher Disease Market Opportunity
- $325 million U.S. market opportunity
- No existing therapies on market or under development
- We address the fatal neurological deterioration of Type 3 disease
- All Type 3 patients should be treated
- ~800 addressable U.S. patient population (100%)
- Although small market, successful companies such as Alexion have focused on diseases with less than 500 patients