Miglustat (Zavesca®) in type 1 Gaucher disease: 5‐year results of a post‐authorisation safety surveillance programme

Miglustat (Zavesca®) is an orally‐available substrate reduction therapy (SRT) for treatment of mild‐to‐moderate type 1 Gaucher disease (GD1) in adult patients unsuitable for enzyme replacement therapy (ERT). Miglustat has not been evaluated in children with GD1, and is not used during pregnancy and breast‐feeding. A non‐interventional, prospective, web‐based safety surveillance programme was initiated at the time of the European launch of miglustat in 2003, and is ongoing. We report the first 5 years of collected data, focusing on neurological manifestations.

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