Why Biogenerics Will Survive Genzyme's Problems

(MoneyWatch)  The Food and Drug Administration rejected Genzyme's request to sell a version of its drug Myozyme made in a new factory, a decision some journalists and bloggers insist on casting as a black mark against the very notion of generic biotech drugs.

There's just one problem: These folks have it exactly backward.

First, some backstory. Genzyme initially won FDA approval for Myozyme, a new treatment for the rare genetic condition Pompe disease, based upon clinical trials that used doses of the drug made in a small test "bioreactor." When Genzyme transferred production to a larger reactor, biochemical tests showed that the new version had a different carbohydrate structure, meaning that it might not be absorbed as readily by muscle cells. Genzyme argued the two versions should be considered equivalent. The FDA disagreed, and now Genzyme has to produce additional human-testing data for the new version to ensure its safety and effectiveness.

What's this have to do with biogenerics? The WSJ Health Blog argues it this way (my emphasis):

The bar likely wouldn't be so high if Myozyme were a typical pill. But the concern with biotech drugs, which are made by living organisms rather than mixing batches of chemicals, is that they are harder to replicate. Biotech companies have been calling for clinical trials to prove that generic versions of brand-name biotech drugs are safe and effective, a higher bar than the typical process for generic approval. That sounds a lot like what the FDA just called for on the product made in the Allston plant, even though that one is from original manufacturer.

Does it? In fact, the FDA's actions here are more or less exactly in line with the way biogenerics proponents would like it to handle copycat biotech drugs -- and pretty much the opposite of restrictions the biotech industry industry has called for in its attempts to protect billion-dollar drugs from generic competition.

The biotech industry has long insisted that any biogeneric drug must undergo lengthy and expensive human testing to ensure its safety and effectiveness. Had the FDA followed such principles in the Genzyme case, there never would have been an issue in the first place, because the company would have been forced to conduct new trials just to qualify its new factory. Instead, the biotech industry has long relied on batteries of simpler tests intended to ensure that new production batches are equivalent to older ones.

(Genzyme isn't the only company to have been tripped up when those tests turn up a difference. In 2001, Genentech also had to conduct new clinical trials to verify that new batches of its psoriasis drug Raptiva -- then called Xanelim -- were equivalent to earlier versions.)

The generics industry has pretty consistently called on the FDA to make such judgments about biogenerics on a case-by-case basis that takes the best possible scientific evidence into account. Now, it's certainly true that biotech-drug molecules are a lot more difficult to characterize than their traditional-drug counterparts -- but that won't always be the case, which is why a case-by-case approach that would allow the FDA to find equivalence based on simpler tests if the evidence warranted makes a great deal of sense.

No one argues that biogenerics won't be a lot harder to make than knockoffs of traditional name-brand drugs. There just doesn't seem to be any reason to order up clinical trials in every case, particularly when the biotech industry itself doesn't bother to do so.

(Photo of a "generic pharmacy" via Flickr user The Consumerist, CC 2.0)