Meanwhile, GTC, a 1993 spin-off of Genzyme, was the first to get a transgenic drug approved. In June 2006, European regulators okayed ATryn, a human antithrombin protein made in goats’ milk, for treating a hereditary blood disorder. Fast-tracked by the agency because it targeted a rare disease, ATryn won FDA approval in February 2009.
Achieving this milestone was hard. Transgenic drug firms had to cope with a complete lack of experience on the part of regulators with the firms’ methods and products, Schnieke says. Early on, she recounts, companies had to help formulate rules on production, quality, and testing. After a decade of work, and just a month before approving the first product, FDA adopted guidelines regulating genetically engineered animals.
Meanwhile, the European Medicines Agency (EMA) is still publishing draft guidelines to ensure the quality of drugs produced in transgenic animals. In guidelines that came out in May, EMA called for vigilance with respect to transgenic-system-specific aspects because “experience with the technology is relatively limited.” Such aspects include animal production and maintenance, as well as product processing, purity, and contamination.
Suitable quality and control systems are needed, according to the guidelines, because standard Good Manufacturing Practices can’t practically be applied to the animal side of the equation. For downstream processing, GMP conditions will apply, EMA says, “since it is expected that a protein that is produced by a transgenic animal, and its quality attributes, would follow the same standards as a protein produced in mammalian cells in a fermentation system.”
EMA approved the second transgenic product, Pharming’s Ruconest, in June 2010. Produced in the mil k of transgenic rabbits, the C1 inhibitor is used to treat hereditary angioedema (HAE) and is sold in Europe by Swedish Orphan Biovitrum. Pharming also filed for FDA approval in late 2010 but was rebuffed. It hopes that results of a Phase III trial now under way will soon support a new application.
If the trial end point is met, Pharming’s North American partner, Santarus, will pay it $10 million. Another $5 million will come if FDA accepts a new filing. Until then, with just over $4 million in the bank, Pharming has been trying to hang on financially and is drawing on up to $13 million in funding from investors. For the first six months of 2012, the firm had revenues of $2.4 million and lost $21 million.