(From The Boston Globe via PharmaLot)
The FDA set an extremely high bar yesterday for biosimilars when it ruled Genzyme could not get clearance to manufacture Myozyme (alglucosidase alfa) to treat Pompe disease (a rare genetic disease that inhibits the breakdown of glycogen in muscles into glucose) at the 2000L scale as opposed to the 160L it currently uses in the US, due to small carbohydrate differences between methods.
The production of Myozyme in the larger reactor has been approved in over 40 countries and in almost 900 patients currently receiving the drug, including the LOTS (Late-Onset Treatment Study) trial, have shown both scales to be safe and effective. The FDA will require Genzyme to submit a new BLA (biologics license application) and market two versions of Myozyme. Genzyme has estimated the delay in approval to reduce EPS by $0.10.
This is an interesting precedent from a few different angles. Genzyme and the FDA agreed in May of 2007 to give Myozyme produced at the 2000L scale away free of charge (thought the Myozyme Temporary Access Program) to approximately 140 patients in the US due to supply issues. So the drug is good enough to give people freely, but not enough to charge for it? It is the job of the FDA to ensure that pharmaceuticals are safe (in relation to the disease) and effective and unless this burden is met, the drug should not be approved. I’m not sure how they can allow patients to be receiving this biologic, while at the same time, asking for more data to support commercialization of Myozyme at this scale.
The discrepancy in the carbohydrate profile of the same enzyme produced in two different scales only underscores the difficulty of producing biologic drugs. If Genzyme can’t copy it’s own product, what hope do Teva or Watson and the like have, especially with such a high burden of proof?
(Image by Siobhan Curran on Flickr under a Creative Commons license)
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Eben is a highly caffeinated business development associate at a small, cash sensitive pharmaceutical company somewhere in Massachusetts. He enjoys cliche-less banter, compartmentalization, non-equilibrium thermodynamics and NPV analysis. Agree or disagree with what he's posted? He encourages comments.
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