Actelion cystic fibrosis drug moves to phase IIa

Actelion has announced the start of a phase IIa clinical trial of miglustat as a treatment for patients with cystic fibrosis.

The drug is being tested in patients affected by a specific cystic fibrosis mutation known as delF508, with a primary endpoint of the effect of the compound on nasal potential difference, measured by tests for the cystic fibrosis transmembrane conductance regulator.

Results from the trial, expected at the end of next year, will dictate the probability and extent of further tests of the compound in this patient population.

Currently, Zavesca (100 mg miglustat) is indicated as an orally-administered treatment for patients with mild-to-moderate forms of type one Gaucher disease for whom it it judged that enzyme replacement therapy is inappropriate.

Jean-Paul Clozel, chief executive officer of the firm, 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.”

He added that this decision also shows the company’s desire to properly explore the pharmaceutical potential of miglustat in a range of potential indications.

In August 2007, Solvay Pharmaceutical reported that Creon (pancrelipase delayed-release capsules, USP) helps meet a medical need for the treatment of pancreatic exocrine insufficiency related to cystic fibrosis.