Zavesca® 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.
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.
In November 2002, we 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, we entered into an agreement with UCB S.A., the legal successor to Oxford GlycoSciences. Under this agreement, Actelion is assigned all of UCB S.A’s rights and obligations regarding miglustat, including marketing rights throughout the world, except in Israel, the West Bank and Gaza Strip. Geographical expansion to Australia, Brazil and Turkey, where we achieved marketing approval in 2007 and early 2008, is further increasing the commercial potential of Zavesca®.
