Who says the FDA isn’t open to new ideas? The U.S. drug agency has given the green-light to GTC Biotherapeutics, a small biotech company in Framingham, MA, to market the first drug in the U.S. ever derived from genetically modified animals.
GTC Biotherapeutics (NASDAQ: GTCB) and partner Ovation Pharmaceuticals of Deerfield, IL, said today that the U.S. drug regulator has given them clearance to begin selling recombinant antithrombin (ATryn) to patients in the U.S. with a rare, genetic blood clotting disorder. The companies expect to introduce the drug to the market before the end of June.
The FDA’s decision comes as little surprise, since a report released last month showed that the agency’s staff considered the drug to be safe and effective, and an outside panel of FDA advisers agreed. This product has generated little in the way of sales for GTC in Europe, where it is currently approved. But more importantly, GTC hopes that the FDA’s decision establishes a clear path for approval of other products made a similar way.
In the case of this drug, a goat is genetically modified to produce more than the usual amount of antithrombin protein, which is secreted through its milk. The proteins are then filtered out from all the other material in goat milk that you wouldn’t want to inject into a person, sort of like how other biotech drugs are purified from fermentation-type techniques. The business attraction of GTC’s method, sometimes called “pharming,” is that it can pump out industrial quantities of protein drugs more cheaply.
“The approval of ATryn marks a significant milestone in the development of this innovative recombinant technology,” said Geoffrey Cox, CEO of GTC Biotherapeutics, in a statement.
The drug’s formal approval covers patients who have a gene mutation that creates a deficiency of antithrombin protein, making them more likely to develop dangerous blood clots. An estimated one out of every 2,000 to 5,000 people in the U.S. has the disorder, GTC says.