Zavesca Trial

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Zavesca Trial

From Actelion, October 27, 2005

A poster presentation at the annual conference of the American Society of Human Genetics (ASH-G) reported one-year results from a randomized controlled study evaluating safety and efficacy of miglustat (Zavesca®) in patients suffering from Niemann Pick Type C (NP-C.

Although not statistically significant, the 29-patient-study showed trends toward improvement or stabilization in terms of saccadic eye movements (by electrophysiological assessment) and swallowing and audition (by clinical assessment) in patients receiving miglustat compared to standard of care. The study will continue as planned for another 12 months, during which time all patients will receive miglustat. A discussion of study findings is contained in: Patterson M., Vecchio D., Prady H., Ait-Aissa N., Abel L., Wraith E. - Poster 2505/T, ASH-G 2005 (Salt Lake City).

Marc Patterson, M.D., FRACP, Professor and Head, Division of Pediatric Neurology at Columbia University New York/USA, commented: "We know already that miglustat crosses the blood-brain barrier and accesses the brain. For the first time, we have been able to observe some potentially beneficial effects in patients with NP-C. I am also encouraged with these observations as they could suggest some restoration of function in neurons that have been altered by the disease process. As such, this study is another step forward in our quest to understand and potentially treat this complex disease."

Twenty-five patients have completed the first 12-month study period and four have discontinued treatment (three in the miglustat group, one in the standard care group). Results from the 12-month data showed improvement in horizontal saccadic eye movements (by blinded centralized assessment) and improvement in swallowing, none reaching formal statistical significance. The analysis also showed stabilization in auditory acuity in treated patients compared with deterioration in the untreated group.

The safety profile of miglustat observed in this study was consistent with the results obtained in patients with GD1 treated with miglustat 100 mg three times daily, particularly regarding weight loss and gastrointestinal disturbances.

Based on these results, the study will continue as planned for a further 12 months during which time all patients will receive miglustat and will be followed with the same set of assessments.