BioWorld International Correspondent
LONDON - Oxford GlycoSciences plc said its lead product, Zavesca, a treatment for Gaucher's disease, received marketing authorization from the European Commission and the company plans to launch the product on the European market in the spring.
The approval of the orally administered treatment is for patients with mild to moderate Type I of the inherited glycolipid storage disorder, for whom the only other treatment, injected enzyme replacement therapy, is unsuitable.
CEO David Ebsworth said getting approval of its first drug was a "major achievement" for the company, based in Abingdon. "With this approval, OGS has succeeded in taking a drug candidate through the development and regulatory processes, to marketing approval."
However, OGS will now hand over the reins to its marketing partner, Actelion Ltd., of Allschwil, Switzerland. The marketing license will be transferred to Actelion, which will be responsible for all regulatory and marketing activities, and for booking sales of Zavesca. OGS said it will be paid a share of net revenues, though the terms of the agreement were not disclosed.
At the same time, OGS and Actelion announced the extension of the European marketing deal, entered into in July, to worldwide rights, excluding Israel.
Ebsworth said that transferring all responsibility for marketing would reduce OGS's cash burn. It also fits in with the recent restructuring of the company, when Zavesca and related programs were split into a separate division. "This extension [of Actelion's marketing rights] moves OGS another step closer to meeting its strategic objective of making its inherited storage disorders business unit profitable by the end of 2005," Ebsworth said.
The partners also said they will jointly submit an amended NDA to the FDA for Zavesca next year. The FDA turned down the drug in June, citing inadequate safety and efficacy data. Following an end-of-review conference, OGS believes it may be possible to get approval for restricted use, as in Europe.
Rather than replacing the missing enzyme glucocerebrosidase, to allow patients to break down glycosphingolipid (GSL), Zavesca inhibits the synthesis of GSL in the first place, by inhibiting glucosylceramide, an enzyme involved in its production.
As well as restricting approval to patients who are unsuited to enzyme replacement therapy, the European authorization also requires follow-up safety data from a post-marketing surveillance program and additional clinical information to be provided on a regular basis.
It was because of these exceptional conditions that OGS chose Actelion as a marketing partner. The Swiss company has similar conditions attached to its product Tracleer, a dual endothelin receptor antagonist for the treatment of pulmonary arterial hypertension. It has set up a web-based pharmacovigilance system for physicians to report any problems. The marketing partnership with Actelion is for five years.
A registration filing for Zavesca also is intended for Japan. The companies have set up a steering committee to evaluate further potential uses of the product in other diseases, such as Type 3 Gaucher's disease, Nieman Pick Type C and late onset Tay-Sachs disease.
OGS's stock (NASDAQ:OGSI) gained 69 cents Tuesday, or 28.6 percent, to close at $3.10.