Zavesca® is 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 NP-C disease, which is a very rare, invariably progressive, and eventually fatal neurodegenerative genetic disorder affecting both children and adults.
Zavesca® (miglustat) is currently the only approved oral treatment for 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.
In November 2002, Actelion in-licensed Zavesca® from Oxford GlycoSciences (UK) Ltd. Market introduction in the European Union began in March 2003, followed by the United States in January 2004. In November 2005, Actelion entered into an agreement with UCB S.A., the legal successor to Oxford GlycoSciences, under which Actelion was assigned all of UCB S.A’s rights and obligations regarding miglustat, including global marketing rights except in Israel, the West Bank and the Gaza Strip.