LONDON - Oxford GlycoSciences plc said Friday it received approval in Europe for Vevesca, an oral treatment for Gaucher's disease that was turned down by the FDA on June 24.
The company also announced a marketing and distribution agreement with the Swiss biotechnology company Actelion Ltd., of Basel, for the product, which in the future will be known as Zavesca because of a commercial conflict over the Vevesca trademark.
OGS CEO David Ebsworth told BioWorld Today, "I'm very happy and very proud for the organization and all the work they've done for the last four years." Ebsworth, who took over as CEO earlier in July, said he "had to get his skates on" to set up the marketing deal with Actelion, which was put together in a few weeks. "I had to be honest, and say the agreement would only be activated with the approval."
The Committee for Proprietary Medicinal Products of the European Agency for the Evaluation of Medicinal Products recommended that marketing authorization be granted under exceptional circumstances, which means the approval is given subject to specific obligations that will be reviewed annually. OGS also is required to provide follow-up safety data from a post-marketing surveillance program and additional clinical information.
Ebsworth said he expected such conditions to become quite normal in Europe for orphan drugs such as Zavesca. "Because of low patient numbers there are going to be cases where the data are not 100 percent clear, but drugs will be allowed on the market where there is clear medical need."
In the case of Zavesca, OGS has data on 80 patients over three years.
It is because of the exceptional conditions that OGS, based in Abingdon, considers Actelion to be such a good 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, and has set up a web-based pharmacovigilance system where clinicians can report any problems.
The FDA said Zavesca was not approvable because OGS had not provided sufficient support for its safety and efficacy. Ebsworth said OGS is still awaiting a date for a meeting with the FDA to review the non-approvable letter.
"I can't speculate if European approval will have any impact," Ebsworth said. "One of the advantages of the European process is that it is iterative: They can come back to you with questions and you can add data. The FDA cuts data with filing; we hope to show the FDA all the data we have shown the Europeans."
OGS's more immediate target in terms of marketing approval is Israel, where a high proportion of the 10,000 or so sufferers of Gaucher's, an inherited glycolipid storage disease, live. Ebsworth said he is going to be in Israel this week to talk to OGS's partners on how to expedite the approvals process there. In April one hospital in Israel suspended trials of Zavesca because of concerns about one patient. But last week that suspension was removed after the ethics committee overseeing the trial reviewed additional clinical data.
Ebsworth said it is not easy to forecast how much revenue OGS will get as a result of the European approval. The approval is limited to the treatment of mild to moderate type 1 Gaucher's disease patients who are unsuitable for enzyme replacement treatment. "In practice, this has included many patients who didn't want injections," he said. "There may be many patients who prefer an oral treatment to a three-hour infusion every two weeks."
Rather than replacing the missing glucocerebrosidase enzyme 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.
The marketing partnership with Actelion is for five years, with Actelion responsible for marketing, sales and post-approval monitoring. OGS will manufacture the drug and remain responsible for regulatory activities. The two will also decide on further development programs for Zavesca, which will be implemented by Actelion.
OGS's shares, which trade on the London and Nasdaq exchanges, have fallen significantly this year due to the ups and downs of the approvals process, but gained on Nasdaq (OGSI) on the news Friday to close at $4.90, up 27 cents.