By Alan Sverdlik
Oxford GlycoSciences plc filed a new drug application in the U.S. last week for its lead product, Vevesca, an oral treatment for Type I Gaucher disease that has shown to be effective as a monotherapy and in combination with intravenous enzymes.
The firm¿s CEO, Michael Kranda, told BioWorld Today that the NDA is a ¿milestone¿ in the history of the Abingdon, UK-based company, which has been trying to establish a product pipeline for its proteomics technology.
¿This demonstrates a real commitment to developing products and moving from a company with a platform technology to a company with a pipeline,¿ Kranda said.
OGS originally had planned to file the NDA application for Vevesca, which has FDA and European Union orphan drug status, by the middle of this year, which the company previously said could have resulted in approval by the end of 2001 and launch as early as the first quarter of 2002.
Kranda would not elaborate on the timetable, but the nearly two-month delay in the application pushes the approval and launching dates back at least two months as well.
Gaucher disease is a glycolipid storage disorder in which an enzyme deficiency leads to the accumulation of unmetabolized lipids. An approved intravenous enzyme therapy named Cerezyme, a product of Cambridge, Mass.-based Genzyme General, replaces the missing enzyme, while Vevesca acts by inhibiting glucosyltransferase, a key enzyme involved in the production of glycolipids, thus cutting the rate of glycolipid synthesis.
¿With enzyme deficiency, there are reduced white cells and platelets and a swelling of the organs,¿ Kranda said.
He said there are several thousand Gaucher patients worldwide and a few hundred doctors who specialize in the disease. Many patients, perhaps half of those with the disease, don¿t have access to treatments.
OGS intends to market Vevesca itself, probably in tablet form, and already has contracted with a manufacturing facility. It is assembling a small sales and marketing force ¿ probably between 20 and 40, Kranda said ¿ that will focus on North America and Europe.
Calling Vevesca ¿a bite-sized sales and marketing opportunity,¿ Kranda added, ¿With the specialty of the patient population, it¿s the ultimate niche market.¿
There have been two key studies that are backing up OGS¿s NDA, both of which in some way compare OGS¿s orally administered treatment with Genzyme¿s intravenous infusion.
The studies showed that ¿you could switch people from intravenous administration to oral administration and maintain them clinically,¿ Kranda said.
One study looked at the impact on patients who went off Cerezyme and on Vevesca, or who had switched to a combination therapy of Cerezyme and Vevesca after being on enzyme therapy for at least two years. That study involved 36 patients. Another, which simply looked at Vevesca as a monotherapy, involved 18 participants.
Kranda said the NDA will include data on 80 patients in all. ¿The data we submit concerns the monotherapy and the switch [study],¿ he said.