Actelion Ltd, Europe's largest biotech company, said on Thursday the U.S. Food and Drug Administration (FDA) would review use of its drug Zavesca to treat a rare neurodegenerative disease early next year.
The FDA has granted priority review for Zavesca, or miglustat, to treat the progressive neurological manifestations in adults and children with Niemann-Pick type C disease (NP-C).
Miglustat could become the first treatment for NP-C in the USA, which would represent a major therapeutic breakthrough for patients and their treating physicians, Actelion Chief Executive Jean-Paul Clozel said.
The Endocrine and Metabolic Drug Advisory Committee will review the supplemental new drug application on Jan 12, 2010, Actelion said in a statement.
Zavesca is already approved for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease in the United States.
Actelion is trying to cut its dependence on key drug Tracleer, which treats a rare heart and lung disorder and rakes in more than $1 billion a year.