BioWorld International Correspondent

Shares in Pharming Group NV rose by about 30 percent over the past two weeks on two disclosures concerning the company's lead drug candidate, recombinant human C1 Inhibitor (rhC1INH).

Last Friday, Pharming said it received fast-track designation from the FDA for the product, which is in development for acute attacks of hereditary angioedema (HAE). A week previously, the Leiden, Netherlands-based company said it filed for European approval for the protein.

The stock closed Monday at €3.81 (US$4.89) on the Euronext Exchange in Amsterdam, up from €2.89 two weeks ago, before the recent disclosures. Analysts expect the compound to gain approval from the London-based European Medicines Agency (EMEA) by the end of 2006 or early 2007. However, with other novel, late-stage products jostling for attention in the space, it is not clear what volume of sales - and what kind of revenue - the company can expect.

Pharming's competitors include Dyax Corp., and its partner Genzyme Corp., both of Cambridge, Mass., who are developing DX-88, a small-molecule inhibitor of kallikrein, an enzyme that catalyzes a key step in the inflammatory cascade associated with HAE. Jerini AG, of Berlin, is developing Icatibant, a bradykinin B2 receptor antagonist, in partnership with Kos Life Sciences Inc., a subsidiary of Kos Pharmaceuticals, of Cranbury, N.J.

Pharming's product, which is produced from the milk of transgenic rabbits, is designed to correct the hereditary C1 inhibitor deficiency directly. Diosynth BV, a subsidiary of Akzo Nobel NV, of Oss, the Netherlands, is responsible for downstream processing and purification of the protein.

It is the second such product in line to gain EMEA approval. In June, GTC Biotherapeutics Inc., of Framingham, Mass., secured a positive recommendation from the agency's Committee for Human Medicinal Products for ATryn, a recombinant human antithrombin produced in transgenic goats, although it still is awaiting formal approval from the European Commission.

Delays in Pharming's pivotal trial of rhC1INH may have damaged the potential of its emerging franchise.

"I think they have lost quite some time. The trial took six months longer than originally planned. Now they are just in the pack with the others. They're not jumping out of it," Christophe Van Vaeck, analyst at Brussels-based KBC Securities NV, told BioWorld International following the company's EMEA filing.

"I think pricing will be quite a dilemma for the three parties," said analyst Bernd Hilhorst, at Amsterdams Effectenkantoor (AEK) BV in Amsterdam. "We have to wait and see how much product they can sell." Nevertheless, the company is in "a very good position," he said. "I think Pharming will be first to launch."

Van Vaeck said there is "significant upside" attached to the stock, because it had weakened because of the general decline in the market and because of the delays associated with the rhC1INH development program.

Pharming officials were not available for comment, but a spokeswoman for the company said the company's U.S. pivotal trial is about six months behind its European program and that it expects to file for FDA approval of rhC1INH around the end of 2006 or the beginning of 2007. Earlier this year, Pharming entered a royalty agreement worth up to $30 million with New York-based Paul Royalty Fund. The company still is seeking marketing partners for rhC1INH in the U.S. and in Northern Europe, the spokeswoman said. A distribution agreement with Barcelona, Spain-based Laboratorios del Dr. Esteve SA covers Spain, Portugal and Greece.