As reported August 9 in The Wall Street Journal, Genzyme has been unable to get FDA approval for its large-scale production of Myozyme, a drug approved last year for Pompe's disease. According to the article, FDA says the product manufactured in the large-scale reactors at the company's Boston facility is not the same as the product used in clinical trials, produced in smaller equipment at the company's site in Framingham, MA. The product manufactured in Boston is approved for sale in 29 other countries, however, and Genzyme maintains it is safe and effective.
Biologics manufacturers are quite familiar with the challenges of scale-up, so this case, though disappointing, is not unique. New this time, however, is how this case is being tied to the debate about follow-on biologics. In a Wall Street Journal blog, journalist David Amstrong says the fact that an experienced company like Genzyme is having trouble manufacturing its own product heightens concerns about the ability of generic manufacturers to copy biotech drugs.But regulatory measures can handle situations like this. Companies regularly file comparability protocols to demonstrate product equivalence following manufacturing improvements or after transferring operations to another site, and Genzyme can use such testing in this case. In the same way, if a biosimilars law is passed in the US, nonclinical testing could be used to support the equivalence of follow-on biologics. In all cases, if such testing appears insufficient, clinical trials should be required. So scale-up challenges like Genzyme's should not be used to raise the spectre of unsafe biosimilars reaching the market.
The other current industry development is Amgen's announcement that it will lay off up to 2,600 workers, following a significant decline in Aranesp sales resulting from safety concerns and reimbursement changes this year. Some financial analysts have said the cause is that Amgen has become too big. It is no longer nimble, they say, and thus is facing the same kinds of problems that Big Pharma has faced—reliance on blockbuster products; decreased R&D productivity; difficulties restocking its pipeline.
It's easy to conclude that Amgen's current challenges are because of the company's size. But, sadly, drug safety issues can happen to any company, large or small. If the same happens to an emerging biotech firm, no one on Wall Street says, "well, they just got too big."
So in analyzing the industry's current challenges, let's be careful before extrapolating about what they mean.
Laura Bush is the editor in chief of BioPharm International,