Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration

This document aims to increase the chances of rapid and smooth agreement of the paediatric investigation plan (EMA)/pediatric study plan (FDA). It also discusses the possibility of a multi-arm, multi-company clinical trial for the treatment of Gaucher disease, as one approach to address the feasibility of developing multiple products for a rare disease in a limited timeframe.

Keywords: Gaucher disease, lysosomal storage disorder, enzyme replacement therapy, substrate-reduction therapy