Actelion receives negative CHMP opinion for Zavesca

Actelion has reported that it has received a negative opinion from the European Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency for Zavesca (miglustat) as a treatment for patients with Niemann Pick type C (NP-C).

Following this announcement, Actelion notes that it will request a re-examination of this decision, with a final opinion on the compound due to be published by CHMP in the first quarter of 2008.

NP-C is a rare genetic condition connected to a number of neurological manifestations, with symptoms caused by the storage of glycosphingolipids in body cells, including brain cells.

Zavesca is currently indicated as an orally-administered treatment for mild-to-moderate type one Guacher disease for whom enzyme replacement therapy is deemed unsuitable.

“The regulatory review in the European Union is primarily based on the 12 and 24-month results from a randomized controlled study evaluating safety and efficacy of miglustat in 29 adult/juvenile patients suffering from [NP-C],” Actelion reports.

The company notes that there currently exists no treatment option for this condition.

Last month, Actelion announced the launch of a phase IIa clinical trial of miglustat as a treatment for patients with cystic fibrosis.