VevescaTM
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 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.