Zavesca

Zavesca(r) is currently the only approved oral treatment for adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is unsuitable. Type 1 Gaucher disease is a rare and debilitating metabolic disorder.

Zavesca(r) (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(r) 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. (Information was obtained from Actelion's website at www.actelion.com).

Status: Available