Techdirt. Stories filed under "fabrazyme"

Fabry Patients Sue Genzyme Over Drug Shortage; After NIH Refuses To Allow Others To Make Fabrazyme

Mike Masnick — Thu, 17 Mar 2011 07:22:59 PDT

One of the more horrifying stories of patents costing lives out there is the situation with Genzyme and Fabrazyme. As we detailed, not enough of the drug is being made allegedly due to manufacturing errors by Genzyme (for which it was fined $175 million by the FDA), and its causing serious health problems -- and even death -- to the sufferers of Fabry disease, who are caught in the middle of this. The patients asked the NIH to allow others to make the drug, via a "march-in" order, so that there would be enough supply, but the NIH refused, basically because it said that the patients (you know, the people actually suffering) couldn't make such a request. The end result, of course, is that lives remain at risk. Genzyme is apparently now rationing the drug, only giving 50% of the recommended dosage, despite a study suggesting that such reduced dosages are a problem:

Neither Genzyme nor Mt. Sinai has tested whether the reduced dosage is either safe or beneficial for treating Fabry disease. In fact, in November 2010, The European Medical Agency determined that the lowered dose of Fabrazyme resulted in patients having more strokes, heart attacks, renal disease and other symptoms including severe GI distress and pain and burning in their extremities (neuropathy). It also determined that the lowered dose actually accelerated the course of the disease in some patients. Europe recommended in October 2010 that all patients be given a full dose of Fabrazyme instead of the lowered dose. Genzyme rejected the European assessment and continued to provide only unapproved low doses to patients in the U.S.

The lawyer for some of those patients, Allen Black, alerts us to the news that his clients have now sued Genzyme and Mt. Sinai Medical School over the continuing shortages (pdf). Mt. Sinai is the actual patent holder, with an exclusive license to Genzyme. The patients are arguing for "compensation and punitive damages for lack of adequate treatment and Genzyme's failure to take adequate precautions including keeping a reserve inventory and providing second sources of manufacturing."

Part of the lawsuit is based on negligence claims -- and given the FDA fine, perhaps there's a basis for a negligence claim. The latter part of the case may be more interesting, however. I'm not sure how much of a chance it actually has, though I certainly sympathize with the plaintiffs. At a high level, it may be tough to show that Genzyme has any legal obligation to make enough of the drug (a moral obligation may be a different story). However, the lawyers make a very key point concerning patent law in making this legal argument: that the Bayh-Dole Act actually gives Genzyme a legal obligation.

The Bayh-Dole Act was designed to help universities better commercialize research in order to get those innovations out to market. For a variety of reasons, I've argued that the law has failed significantly in this regard, but these patients are picking up on a key part of Bayh-Dole, which is that the law was put in place to prevent patentees from denying citizens access to publicly funded inventions. It thus may require that certain standards be met when it comes to federally funded inventions -- which Fabrazyme certainly is. The law specifically says that it was put in place to "protect the public against nonuse or unreasonable use of inventions." And since the plaintiffs feel that this is the case here, they're arguing that they're now able to recover damages (though you'd have to imagine they'd prefer full doses). If that argument succeeds, it could lead to a very interesting series of follow-up cases concerning other federally-funded inventions.

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NIH Won't Let Others Supply Life Saving Drug Even Though Genzyme Can't Make Enough

Mike Masnick — Wed, 8 Dec 2010 07:21:13 PST

Earlier this year, we wrote about the situation with a petition to the National Institute of Health (NIH) to let other companies produce Fabrazyme, a drug that treats Fabry disease. Fabry disease is an enzyme deficiency that can create very serious problems in those who have it -- including kidney failure and heart attacks. Genzyme holds the patent on Fabrazyme, but has a problem: it can't produce enough of the drug. That means people die. A group of Fabry patients petitioned the NIH to see if other companies could be allowed to produce just a little more Fabrazyme. They didn't ask to break the patent. All they wanted was to let others make the drug, and pay Genzyme a royalty. On top of this, it's worth noting that the key research for the development for Fabrazyme wasn't actually done by Genzyme, but was done by the Mount Sinai School of Medicine and was financed with taxpayer money from the NIH.

At the time, we doubted the petition would be approved -- and indeed, the NIH has rejected the petition. An opening was left for an appeal, if another petition is filed by a company that wants to make Fabrazyme, rather than the patients (you know, the folks actually suffering). As the statement of Robert Weissman from Public Citizen makes clear, this is a ridiculous situation:

Why is NIH resorting to legal gymnastics to avoid exercising its legal authority to ensure an adequate supply of an important medicine? NIH agrees Genzyme is failing to produce an adequate supply of the important medicine Fabrazyme. It is plain that removing the patent monopoly on the drug is a necessary condition to enabling other potential manufacturers to enter the market. Yet NIH chooses to deny a request to issue open licenses for the Fabrazyme patents -- a request for which it has undisputed legal authority -- on the Alice-in-Wonderland, self-justifying grounds that there are as yet no competing suppliers. Of course there are no competing suppliers -- why would any firm try to enter a market it believed closed by a patent monopoly?

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Patents Getting In The Way Of Saving Lives; Fabry Disease Sufferers Petition US Gov't To Step In

Mike Masnick — Tue, 3 Aug 2010 12:52:00 PDT

Whenever I read stories like this one, it just makes me wonder how people can defend patents in certain situations. Genzyme is a pharma firm that has a patent on a drug, Fabrazyme, which is used to treat Fabry disease, an enzyme deficiency that can create very serious problems in those who have it -- including kidney failure and heart attacks. The problem? Genzyme apparently can't produce the supply needed by patients. Now, in a true free market, when supply was less than demand, a competitor would step up production, but (oh wait!) there can't be any competitor, because the patent means that Genzyme is currently the only one legally allowed to make the drug. Now a group of patients who have been forced to ration their dosage at one-third the usual amounts, leading to serious health problems and at least one death, has petitioned the government for the right to break the patent.

They're not trying to completely strip Genzyme of the patent. They're merely asking the government to let others produce the drug, and pay Genzyme a mandated 5% royalty. Now, I know the typical response from patent system supporters: without the patent, perhaps this drug wouldn't even exist. The only problem with that is that it's almost certainly not true. The actual research for Fabrazyme was actually done by the Mount Sinai School of Medicine financed by the National Institute of Health. Yes, you read that right. This drug was discovered with taxpayer money... but they were still able to get a patent and then license it to Genzyme.

Chances are this petition won't be approved. These petitions are never approved. But it does highlight the ridiculousness of the current patent system potentially putting people at risk. It's a travesty that federally funded research has been locked up under a patent and limited to only one producer, leading not only to very high prices for the drug, but also excessively limited supply that is putting people lives in danger, and may have already killed one person.

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