Actelion starts miglustat Phase II study for cystic fibrosis

This study will treat 25 patients affected by a specific cystic fibrosis (CF) mutation (delF508). As a primary endpoint the study will investigate the effect of miglustat on the nasal potential difference, a sensitive and non-invasive functional test for the cystic fibrosis transmembrane conductance regulator (CFTR). Defects of CFTR are responsible for the characteristic morbidities of the disease.

Actelion expects full results of this proof-of-concept clinical study to become available at the end of 2008. These results, if positive, will determine the need, size and duration of future studies.

Jean-Paul Clozel, CEO, said: “Actelion’s decision to engage in cystic fibrosis with miglustat reinforces the company’s commitment to rare genetic diseases with significant unmet medical needs. It also reflects our interest to fully explore the potential of the unique pharmacological properties of miglustat for new indications.”