Biotechnology Healthcare - November/December 2008 - (Page 27) Creating a Pathway Over the Regulatory Chasm: A Fresh Start With the election over, a clear framework for guiding follow-on biologics to market may soon emerge. But difficult issues of cost, timing, and even terminology are keeping innovators and generic rivals on opposite sides of the fence. BY ED SILVERMAN L JOHN S. DYKES/CORBIS ast spring, Alison Lawton, senior vice president of regulatory affairs at Genzyme, received surprising news from the U.S. Food and Drug Administration. Rather than approve a largerscale version of the company’s own alglucosidase (Myozyme), a biologic used to treat Pompe disease, the agency decided that the drugmaker would have to submit a separate biologics license application. The reason? The FDA ruled that a larger-scale version made at a different manufacturing site should be considered a different product, because of small differences in chemical structure. The move underscored the complicated debate over the process to be used by regulators to approve follow-on biologics. The FDA’s decision in the Genzyme case clearly suggested the agency’s reluctance to approve follow-on biologics, sometimes called biosimilars, without sufficient clinical data to prove that the new version is at least as safe and effective as the original — even if there are only slight differences in compounds from the same manufacturer. In other words, the decision exemplified the case-by-case approach the agency is taking toward follow-on biologics, even in a situation where the same company makes both versions of a treatment. “Was this a [case of a] new company trying to understand the product? No. We’re the innovator. We have a tremendous amount of experience with this product. Not just the manufacturing, but understanding the biochemical structure and activity,” says Lawton. “We identified small differences in the carbo hydrate structure. We provided a lot of information — we provided additional clinical data, although the data were limited because the number of patients was small, given that this is a rare disease. And the FDA struggled with whether this was sufficient. In the end, they weren’t NOVEMBER/DECEMBER 2008 · BIOTECHNOLOGY HEALTHCARE 27
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