[Ip-health] Pharmalot: Patients Sue Genzyme & Mt. Sinai Over Fabrazyme

Thiru Balasubramaniam thiru at keionline.org
Fri Mar 11 04:24:34 PST 2011


http://www.pharmalot.com/2011/03/patients-sue-genzyme-mt-sinai-over-fabrazyme/

Patients Sue Genzyme & Mt. Sinai Over Fabrazyme
By Ed Silverman // March 10th, 2011 // 3:33 pm


A half dozen people who suffer from Fabry disease have filed a lawsuit  
against Genzyme and Mt. Sinai Medical School for the way ongoing  
shortages of the Fabrazyme treatment is being handled. The med is the  
only treatment approved by the FDA for the rare, but life-threatening  
genetic disease, but Genzyme is rationing due to long-running  
production problems.

Specifically, the biotech imposed a rationing system in which patients  
receive only half of the approved dosage, while newly diagnosed  
patients are prevented from receiving Fabrazyme at all. The move came  
after Genzyme encountered severe manufacturing problems that began in  
2009, prompting a $175 million fine, a consent decree and, more  
recently, a $20 billion takeover by Sanofi-Aventis.

Meanwhile, as many as three Fabry patients have died as a result of  
the rationing, according to the lawsuit, which seeks class action  
status and names Mt. Sinai because the medical center licensed  
Fabrazyme to Genzyme and went along with the rationing plan. The  
lawsuit also notes that neither Genzyme or Mt. Sinai, which is based  
in New York, has tested whether the reduced dosage is safe or  
effective for treating Fabry disease. A Genzyme spokeswoman declined  
to comment.

The suit cites a recent finding by the European Medical Agency that  
the lower dose caused more strokes, heart attacks and renal disease  
and neuropathy, among other problems, in patients. In some cases, a  
lower dose accelerated the course of the disease. The EMA recommended  
a full dose (see this), but the suit claims Genzyme has not told Fabry  
patients of the EMA study results.

And in an interesting maneuver, the Fabry patients want the court to  
decide whether patients who are guaranteed access to tax-payer funded  
inventions under the Bayh-Dole act may recover individual damages  
under the statute when a contractor misuses the invention or withholds  
it from the public. A Mt. Sinai researcher developed Fabrazyme with a  
grant from the National Institutes of Health. While Fabrazyme is  
covered by a patent, the Fabry patients argue the Bayh-Dole Act  
requires protection against the non-use or unreasonable use of  
inventions paid for with tax dollars.

Some of the same patients, by the way, recently petitioned the FDA to  
insist that overseas stock of the med is first made available to US  
citizens (back story). They had previously tried unsuccessfully to  
convince the NIH to override the Fabrazyme patent in a bid to find  
another means of production (read this).

“Genzyme originally told these patients almost two years ago that  
there would be no harm in missing treatments and that the problem  
would be fixed in less than a month,” Allen Black, one of their  
lawyers, says in a statement. “Instead, I am seeing my clients die  
right before my eyes. But the really tragic part of this story is that  
a treatment was available the whole time. However, because there are  
no safeguards or deterrents for such irresponsible behavior, the  
patients must bear the injuries and cost of the shortage, not the  
company that caused it.

“It defies belief that, even though US tax dollars paid for the  
invention of the drug and its development, American citizens are now  
told that they must suffer and die while paying almost $240,000 a year  
for a dose of drug that is untested, dangerous, and too low to even  
help them. Worse, some of my clients were diagnosed after June 2009,  
so Genzyme simply bans those Americans from receiving any drug at all.”


------------------------------------------------------------


Thiru Balasubramaniam
Geneva Representative
Knowledge Ecology International (KEI)
thiru at keionline.org


Tel: +41 22 791 6727
Mobile: +41 76 508 0997








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