GTC sees a silver lining in rejection
Back in 1997, executives at GTC Biotherapeutics — then called Genzyme Transgenics — predicted the company would commercially launch the world’s first drug produced in the milk of genetically-engineered animals within a few years. The company’s transgenic goat herd, supposedly able to produce disease-fighting proteins more efficiently than multi-million-dollar pharmaceutical factories, was expected to revolutionize the drug industry from the reaches of its innocuous-looking Charlton farmstead.
Nearly 10 years and hundreds of millions of investor dollars later, Framingham-based GTC recently suffered a near miss in its bid for regulatory approval of its first transgenic drug. But company spokesman Thomas Newberry says there is reason for optimism that the little biotech company’s long road to validation is nearing a successful end.Newberry admits GTC had expected that Europe’s top drug-regulation committee — the Committee for Medicinal Products for Human Use — would approve his company’s anticlotting compound, ATryn, last February. The company had submitted the trial — for a transgenically produced form of antithrombin — in response to the European agency’s call for such candidates. Aimed at treating a very limited patient population, those with an inherited deficiency of the blood plasma protein that controls blood clotting, the study was small in scope. The regulatory specifications only called for 12 patients to be studied. While the resulting commercial market would also be small, Newberry says it seemed a good strategy for a small company like GTC to get an initial approval for a drug produced by putting human genes into an animal, which then produces a desired human protein in its milk.
From that first small commercial application, GTC could begin making inroads into larger drug markets. Pharmaceutical companies, some of which have partnered with GTC over its fifteen years of research but shied away from embracing the technology, would finally see regulatory validation.
The strategy backfired, however, when CHMP rejected ATryn based on insufficient clinical data. Regulators decided to exclude nine of the 14 patients studied because those patients, pregnant women using the antithrombin therapy to prevent life-threatening clotting during child birth, had to have multiple blood tests to insure desired levels of the protein. CHMP then concluded the sampling was insufficient for approval.
GTC is appealing the ruling and will know in June if CHMP changes its mind. But Newberry says, regardless of the appeal outcome, the ruling represents a victory for GTC’s technology and the company will move forward on other fronts.
The biggest plus, Newberry says, is the fact that regulators fully evaluated GTC’s goat farm and the process it used to produce ATryn and had no concerns with its transgenic technology.
GTC is enrolling 31 subjects, including the nine pregnant patients, for a U.S. Phase III trial of ATryn and is hoping for FDA approval by 2008.
The company will also move forward with a $73-million partnership with LEO Pharma A/S of Denmark to market ATryn in Europe for patients with acquired antithrombin deficiency, a much larger commercial market.
Phillip Nadeau, an analyst at SG Cowen & Co. who follows GTC, agrees that there is reason for optimism. There is no reason to think that U.S. regulators will reject ATryn, he says, though he terms the company’s appeal of the European ruling "a long shot."
Micky Baca can be reached at mbaca@wbjournal.com
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