Zavesca® (100 mg miglustat) is the first and only oral medication approved for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease, and it may only be used in those patients for whom enzyme replacement therapy is unsuitable.

Zavesca® (miglustat) is also approved in the European Union for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C disease (NP-C). Zavesca® is the first treatment to be approved for patients with Niemann-Pick type C disease, a very rare, invariably progressive and eventually fatal neurodegenerative genetic disorder affecting both children and adults.