Actelion drops EMEA indication extension for Zavesca

The European Medicines Agency (EMEA) says it has been formally notified by Swiss drug firm Actelion of its decision to withdraw the application for an extension of indication for the centrally-authorized drug Zavesca (miglustat).

Zavesca, an orphan medicinal product, was expected to be used for the treatment of neurological manifestations in patients with Niemann Pick type C disease, a rare inherited neurodegenerative disease of childhood and adolescence. It was first authorized in the European Union in November 2002 and is currently indicated for the oral treatment of mild to moderate type 1 Gaucher disease. Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. The drug generated revenues of 35.3 million Swiss francs ($32.0 million) in 2007, up 38% on the previous year.

The application for the extension of indication for Zavesca was submitted to the EMEA on October 16, 2006. The Agency's Committee for Medicinal Products for Human Use (CHMP) had given a negative opinion recommending the refusal of the type II variation to extend the indication last October. The company had requested a re-examination of the negative opinion, which had not been completed when the company withdrew, says the EMEA.