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."