BioWorld International Correspondent
LONDON - Oxford GlycoSciences plc (OGS) said it will submit a new drug application in the U.S. by the middle of 2001 for its lead product, Vevesca, an oral treatment for type 1 Gaucher disease.
That disclosure followed preliminary analysis of a six-month study showing equivalence between Vevesca and intravenously administered Cerezyme enzyme therapy, a Genzyme General product. Twenty-nine of the 36 patients in the trial decided to switch to Vevesca when the study ended.
OGS Chief Financial Officer Stephen Parker told BioWorld International, "These patients elected to continue with Vevesca before we had the results of the medical assessments, although we knew from the previous monotherapy trial that Vevesca is as effective [as Cerezyme] over the same endpoints."
Vevesca has FDA and Europe Union orphan drug status, and Parker estimates the fast-track designation should lead to approval around the end of 2001. "We hope in the U.S. at least we might well be launching in the first quarter of 2002."
There are fewer than 100 doctors worldwide specializing in Gaucher disease and OGS intends to market the product itself. It already has established a manufacturing facility.
Gaucher disease is a glycolipid storage disorder in which an enzyme deficiency leads to the accumulation of unmetabolized lipids. While Cerezyme replaces the missing enzyme, Vevesca acts by inhibiting glucosyltransferase, a key enzyme involved in the production of glycolipids, and thus cutting the rate of glycolipid synthesis.
The objective of the study was to investigate oral treatment with Vevesca in patients who had been receiving Cerezyme by intravenous infusion for at least two years. Twelve switched to Vevesca, 12 received Vevesca in addition to intravenous enzyme and 12 remained on enzyme therapy. Liver and spleen volumes were assessed by computerized tomography at the start and end of the trial, and hematological and biochemical parameters were measured.
Thirty-three patients completed the trial. The initial results indicate that patients were successfully maintained on oral therapy alone. "Patients preferred the oral treatment, and the results show it is as effective," said Parker.
OGS, based in Abingdon, Oxfordshire, said preliminary data from a further six-month monotherapy trial in 18 patients, which has just been completed, also demonstrated the clinical benefit of Vevesca. The NDA will include data on 80 patients, including 14 who have been treated for two years.