Comments on: FDA Decision a Blow For Genzyme—and Biogenerics? http://www.xconomy.com/boston/2008/04/22/fda-decision-a-blow-for-genzyme-and-biogenerics/ Business + Technology in the Exponential Economy Sun, 22 Nov 2009 19:43:29 -0600 http://wordpress.org/?v=2.8.6 hourly 1 By: Ronald A. Rader http://www.xconomy.com/boston/2008/04/22/fda-decision-a-blow-for-genzyme-and-biogenerics/comment-page-1/#comment-14158 Ronald A. Rader Wed, 23 Apr 2008 16:37:31 +0000 http://www.xconomy.com/2008/04/22/fda-decision-a-blow-for-genzyme-and-biogenerics/#comment-14158 This may not be as bad an omen for follow-on biologics/biosimilars in the the U.S. than is being portrayed by many. Nothing of substance has yet been disclosed, e.g., what are the structural and, perhaps, functional differences between enzyme manufactured at 160- and 2000-L scales. Obviously, there are at least some detectable structural differences for FDA to require a full BLA vs. supplemental filing. The problem is, presumably, that the products produced at two different scales, in two different facilities, etc. have been portrayed by Genzyme as being identical, interchangeable or truly (bio)generic. FDA has already stated that it essentially will not even consider such biologics approvals for years. Genzyme probably would have had a much easier time, and the new product already be approved, if it had developed the 2000-Liter-scale product much like a follow-on biologic or biosimilar would be, i.e., had conducted comparison trials and shown bioequivalence. If this were proven, even with some trivial structural differences (not affecting functionality), presumably the new product would simply have received a supplemental approval. I could be wrong, but I am not aware that Genzyme conducted such comparison trials. Another problem (from FDA's perspective) is that Phase III-like trials meeting their primary endpoints for efficacy and safety with the new product have already been completed and reported (but not submitted). Between having full (vs. supplemental) approval-supporting trial data available (but not filed) and with some obvious detectable differences between the products, FDA presumably feels compelled to request Genzyme go for full approval as a new/different product. Ronald A. Rader Author:  Biopharmaceutical Products in the U.S. and European Markets E-mail:  biotech@biopharma.com Web site:  www.biopharma.com This may not be as bad an omen for follow-on biologics/biosimilars in the the U.S. than is being portrayed by many. Nothing of substance has yet been disclosed, e.g., what are the structural and, perhaps, functional differences between enzyme manufactured at 160- and 2000-L scales. Obviously, there are at least some detectable structural differences for FDA to require a full BLA vs. supplemental filing.
The problem is, presumably, that the products produced at two different scales, in two different facilities, etc. have been portrayed by Genzyme as being identical, interchangeable or truly (bio)generic. FDA has already stated that it essentially will not even consider such biologics approvals for years. Genzyme probably would have had a much easier time, and the new product already be approved, if it had developed the 2000-Liter-scale product much like a follow-on biologic or biosimilar would be, i.e., had conducted comparison trials and shown bioequivalence. If this were proven, even with some trivial structural differences (not affecting functionality), presumably the new product would simply have received a supplemental approval. I could be wrong, but I am not aware that Genzyme conducted such comparison trials. Another problem (from FDA’s perspective) is that Phase III-like trials meeting their primary endpoints for efficacy and safety with the new product have already been completed and reported (but not submitted). Between having full (vs. supplemental) approval-supporting trial data available (but not filed) and with some obvious detectable differences between the products, FDA presumably feels compelled to request Genzyme go for full approval as a new/different product.

Ronald A. Rader
Author:  Biopharmaceutical Products in the U.S. and European Markets
E-mail:  biotech@biopharma.com
Web site:  http://www.biopharma.com

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