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

Current version

PDF iconAdopted collaborative approach

Reference numberEMA/237265/2017
Published30/06/2017
KeywordsGaucher disease, lysosomal storage disorder, enzyme replacement therapy, substrate-reduction therapy
DescriptionThis 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.


Document history

First version

Current version

PDF iconAdopted collaborative approach


PDF iconOverview of comments


PDF iconDraft collaborative approach

Published: 30/06/2017

In operation: 30/06/2017-present


Published: 30/06/2017


Published: 14/05/2014

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