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."
13 Comments
Great scoop on this suit. This really is anger-provoking. These people waited a decade longer than they should have because of Genzyme and Mt. Sinai dithering (see my book), and now, because they don't have their manufacturing act together, people allegedly died. If these allegations are proven true, a prosecutor should go after the executives responsible.
I can understand litigous individuals attempting to sue Genzyme.... But Mt. Sanai as well? Lawsuits like these stifle the abilities of institutions to be innovative and provide an environment for research. It is lawsuit abuses such as this that will ensure that others with rare diseases will never get the treatments that they are waiting for. It is much easier to allow greed to win out rather than take on the task of changing the system that creates the current environment. Do not forget that what you claim is perfectly legal per your legislators. Oh well, I guess money is always superior to morality. How sad.....
To address Mel, innovation and ensuring a reliable drug supply go hand in hand-- you must accomplish both to save lives. Obviously, a child that is denied access to a life-saving drug by a shortage will die just as surely as if a drug company never invested in the first place. Nobody should have to choose between a having a reliable drug supply and promoting innovation. They are simply not mutually exclusive.
Also, Mt. Sinai is currently suing the only other manufacturer of a treatment for Fabry disease (Shire's Replagal) in Germany and Sweden, even though there is a world-wide shortage. The amount of money Mt. Sinai wishes to recover against Shire is substantial.
Genzyme has enough drug now to treat all the Fabry patients. The new production facility in Allston is currently operational and producing Fabrazyme. Genzyme cannot release this drug to patients without FDA authorization, and the FDA has not inspected the new, fully functional plant.
It is the FDA that is keeping us on low rations. Only the FDA can get us the drug we need. Suing Genzyme is futile.
Neither the FDA or Genzyme has indicated that Lisa's statement is true. If the comment has any validity please post the source.
Lisa, How do you know Genzyme has the drug? How long does it take for them to be inspected? I just spoke to my MD today and he says there will be half of my dose for the month of March, and half of my normal dose for April. Please let us know if you have more information.
We would really be interested in additional fact concerning the statement by Lisa that the FDA is holding up distribution of the drug. If so,we all need to contact our Congressional Reps. and request pressure be placed on the FDA. If that is not the case, then all Fabry patient's need to be named in a stronger class action suit. It is tragic that these patients are made to suffer further disabilities and even death resulting from the POOR PERFORMANCE of Genzyme. The responsible parties need to be made accountable and additional companies need to be allowed to manufacture this life sustaining drug. These patients are real human beings with vital roles in their families. They deserve nothing less!
I as one of the " lab rats " that did allow the Genzyne Co. to perform tests on myself and my ( at the time ) infant son, Kyle Willink. We stayed at Mt. Sinia as inpatient for an entire week of taking blood, skin test, eye test, urine test,... for the simple reason I as a Fabrys patient along with my Fabrys patient infant son-would have hope of a treatment for this rare disease. So we are good for Genyzme when we were their "lab rats"-and have taken the treatment-as my son was the 1st pediatric patient to receive this drug-but now to be dropped off the chart from Genzyme Co-I WAS TOLD WE WOULD BE OF THE FIRST TO TAKE THIS TREATMENT -AS WE ALLOWED GENZYME TO USE US AS A LAB RATS TO NOT ONLY HELP OURSELVES BUT OTHER FABRYS PATIENT!! GENZYME BROKE THEIR VERBAL CONTRACT WITH US ALL IN THE NAME OF GREED!!
GOD BLESS
LAURA BROWN
I am a Fabry Patient who like the rest of us has been on a reduced amount of Fabrazyme for over two years. I wanted to get information on how to go about joining a Clsss Action Lawsuit against Genzyme Corporation.
i am one of the fabry patients who is in dire pain everyday and fatigue all the time. i just want genzyme to be honest and give us the medicine we all deserve. I can no longer work because of this issue and my husband has been sole provider for me and our kids. This is very depressing and hard. any lawsuit that will get us the medicine we need
Regarding Lisa's claim, I was told the SAME THING by my Genzyme Caseworker. The FDA has not inspected the facility and/or drug and therefor they cannot release the drug in possession. But Im sure next month will be a new and different story.
Bill, Did you ever get any information on how to join the Class Action Lawsuit? Two years is long enough to be be patient. I am so sick of getting letters from Genzyme saying "sorry for the inconvenience" like it's an interruption in a magazine subscription or something. Morally, they owe us more of an answer than just "there will be a delay in shipment." This is changing the quality of life for us all. Thank you, Angela
Where was Genzymes back up plan. No stock piling, no secondary facilities yet the company was compiling increadible amounts of money to put in their pockets. Dont tell me about unnessesary litigation unless you have walked in my shoes. You have no idea what Fabry disease will do to a person, a family and a community. You should be ashamed of your yourself. Picture yourself going to the pharmacy and each and every time you tryed to get a prescription filled you were told sorry we're out of that. You woud be pissed off wanting retribution yet you tell thousands of Fabry patients to hold their toungue when the only drug to save their lives is unavailable for over 2 years. FU. I invite you to come and let me take my pound of flesh from you then.