Vevesca is a potent inhibitor of glucosylceramide glucosyltransferase, the enzyme responsible for converting ceramide to glucosylceramide during GSL biosynthesis. A wide variety of preclinical studies have demonstrated that Vevesca provides an effective control over the rate of GSL synthesis.
OGS is investigating whether Vevesca can be used to treat patients with Gaucher's disease. The goal of this treatment is to employ Vevesca to re-balance the rate of synthesis against the impaired rate of GSL degradation in the cells of these patients. In particular, OGS is determining whether Vevesca can be applied as a monotherapy and is also exploring the combination of Vevesca with replacement enzyme.
In contrast to the approved Gaucher's disease therapy which require intravenous infusions, Vevesca is under clinical trials as an oral therapy.


Progress with Vevesca

Oxford GlycoSciences  has completed preliminary analysis of its six month study to investigate the potential of oral treatment with Vevesca in patients with Gaucher disease who have been receiving enzyme therapy (CerezymeŽ) ('enzyme'). The initial results indicate that patients were successfully maintained on oral therapy alone during the study period.

Based on a review of the data, OGS is planning to submit its New Drug Application (NDA) for Vevesca to the US Food and Drug Administration (FDA) in mid 2001 and to submit a Marketing Authorisation Application (MAA) in Europe on a similar schedule. Vevesca received 'fast track' designation from the FDA in June 2000. The regulatory package is expected to include data on up to 80 patients with type 1 Gaucher disease who have received Vevesca, including 14 patients who have been treated for two years.