A Possible Bump in the Road for the Sanofi-Genzyme Deal: Patients Sue Genzyme Over Fabrazyme Shortages
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The almost two-year manufacturing woes of orphan drug manufacturer Genzyme have been well documented and publicized. Despite these problems and an FDA consent decreed, it was not enough to stop Sanofi-Aventis from doggedly pursuing Genzyme as a take over target for the past year.
As you may recall, production shortages of several of Genyzme’s key products, most notably Fabrazyme, a treatment for Fabry disease (a rare genetically inherited lysosomal enzyme storage disease) forced Genzyme to ration Fabrazyme to patients. Fabrazyme is the only approved treatment for Fabry disease and the rationing plan called for patients already taking Fabrazyme to receive half the approved dosage while newly diagnosed patients were prevented from receiving the drug at all!
A post on the Pharmalot blog last week revealed that at least half a dozen patients with Fabry disease who were taking Fabrazyme filed a lawsuit against Genzyme and New York’s Mt. Sinai Medical School for the ongoing shortages and the ill-conceived rationing plan. According to the lawsuit, as many as three patients with Fabry disease have died as a result of the Genzyme rationing plan. Mt. Sinai was named as a co-defendant in the case because it licensed Fabrazyme to Genzyme and went along with the company’s rationing program. Further, the lawsuit contends that neither Genzyme nor Mt. Sinai had adequately tested whether or not the reduced dosage was effective as a treatment for Fabry disease. Obviously, the FDA approved Fabrazyme as a treatment for Fabry disease based on the dosage information that Genzyme determined to be optimal in it BLA.
The lawsuit will likely never make it to trial (Genzyme will undoubtedly settle) and therefore have little or no impact on the impending acquisition of Genzyme by Sanofi-Aventis. Companies that are being acquired or merging don’t like it much when there is outstanding litigation that may interfere with the transaction.
Nevertheless, Genzyme’s manufacturing problems highlights one of the weaknesses of the US Orphan Drug Act. For those of you who may not know companies granted approval of drugs for orphan indications (< 200,000 patients) are guaranteed seven years of market exclusively (along with tax credits, grants and less onerous clinical trials). This means that no other company (aside from the innovator company) will be granted regulatory approval for a similar orphan product for seven years from the approval date of the first product. Not surprisingly, this forces patients with orphan diseases to exclusively rely on a single drug that is manufactured by a single company i.e. there is no backup. And, if an orphan drug manufacturer has regulatory compliance issues or goes out of business etc the patients that rely on the drug are SOL (as it is said in the vernacular). Unfortunately, this was exactly what happen with Fabrazyme when Genzyme’s chronic manufacturing problems resulted in shortages of the drug.
While many people take drug companies to task for developing so-called “me too” drugs there is a reason why FDA and other regulatory agencies approve them. That is: when there is more than one manufacturer of drugs that treat a particular indication then there will be alternate treatments available to patients if something goes awry with one or another of the products. Although I am a strong proponent of the Orphan Drug Act, the recent entry of several major pharmaceutical companies like Pfizer, GlaxoSmithKline and others into the orphan drug development space suggests that the seven years of market exclusivity offered by the Act may no longer be necessary. Further, shortening or eliminated the market exclusivity term would like help to increase competition among orphan drug developers. Increased competition would, in turn, and then help to drive down the price of orphan drugs which are currently exorbitantly expensive and sometimes not covered by insurers. Changing the orphan drug act would primarily benefit patients with rare diseases and not drug makers. And, in the end, helping patients is admittedly all that matters!
Until next time...
Good Luck and Good Job Hunting!!!!!!!!
After a five month-long series of very public and often acrimonious negotiations, it appears that Sanofi-Aventis and Genzyme may be close to deal that would enable the French drug maker to acquire one of the world’s largest public biotechnology companies.
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Today's New York Times reported that the US Food and Drug Administration (FDA) issued warning letters and ordered 14 pharmaceutical and biotechnology companies to stop running what it calls misleading ads on internet search pages displayed by search engines like Google. The agency faulted the companies for failing to identify product names (brand) and not listing potential side effects (only benefits) for the drugs. In other words, the ads lacked “fair balance” something that FDA stresses and that all drug makers are very familiar with. 
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About a year ago, I was eating lunch and bunch of pharma executives were at the table next to me. I inadvertently overhead bits of their conversation and I heard the words, Facebook, MySpace and YouTube mentioned. This suggested to me that pharma was more aware of social media (and its business implications) than pharma publicly cared to admit. Pharma has been reluctant to embrace social media because of possible legal and regulatory ramifications. Nevertheless, a few companies have decided to boldly go where no pharma company has gone before—to YouTube.
Astra Zeneca
As many of you know, the pharmaceutical industry has been trending downward for the past year or so. Weak pipelines, uncontrolled corporate expansion and soaring drug prices have been offered to explain the recent down turn. However, the real back story to the downturn is the loss of future revenues that is expected to occur starting in 2010 when many current blockbuster pharmaceutical products, 
According to 