[Ip-health] Fabry Patients Sue Genzyme over Drug Shortage

Allen Black drallenblack at verizon.net
Sun Mar 13 12:23:23 PDT 2011

Hi Jamie,
I wanted to update folks on our latest effort.  The news release is below.
We were also picked up on Pharmalot (
e/) and Patent Docs
Best regards,

Genzyme Corp.-  Genzyme and Mt. Sinai Medical School sued for withholding
Fabrazyme®, a taxpayer developed drug, from Fabry Disease Patients

Pittsburgh, PA – March 10, 2011, -- Today, Fabry disease victims sued
Genzyme Corporation and Mt. Sinai Medical School for rationing some patients
and banning others from receiving the life-saving drug, Fabrazyme®.  The
drug is the only approved FDA treatment for Fabry disease, a relatively rare
but life-threatening genetic disease.  Currently, Genzyme rations patients
to only 50% of the FDA approved dosage and bans new patients from receiving
the drug.  

Rationing began almost two years ago, and since then, patients have had a
return of severe symptoms are increasingly likely to die.  As many as three
Fabry patients are reported to have already died during the Genzyme
Rationing Plan.  The patent to manufacture Fabrazyme® is held by Mt. Sinai
Medical School and was obtained as a result of public funding by the
National Institutes of Health under the Bayh-Dole Act.

A copy of the complaint is available on the web at

Genzyme, which produces Fabrazyme® under an exclusive license from Mt.
Sinai, has been unable to produce enough drug to treat the US Fabry disease
market since June of 2009 due to multiple serious manufacturing errors
including viral contamination and contamination of vials with glass, steel,
and rubber particles, which resulted in an FDA consent decree fine of $175
million dollars.  Genzyme instituted its rationing plan to reduce the dose
given to existing patients to as little as 1/3 of the FDA approved dose,
while also banning newly diagnosed patients receiving any drug at all.
Genzyme made over $400 million dollars a year from Fabrazyme® before the

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. 

Due to their injuries during the ongoing Genzyme Rationing Plan, Fabry
patients request 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.
The case is presented by six named Fabry disease patients and as a class

Also for the first time, the courts will be asked to determine 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. Even
though Fabrazyme® is covered by a patent, taxpayer dollars paid for it under
the Bayh-Dole act (35 U.S.C. § 200 et seq.).  The Bayh-Dole Act specifically
requires that the public be protected against a patentee’s nonuse or
unreasonable use of inventions paid for with tax dollars.  

Fabry disease is a rare disorder with an estimated prevalence in the general
population of 1 in 117,000 people, with as many as 1,500 or more patients in
the U.S alone.  Those with the disease are unable to metabolize fats
properly leading to numerous symptoms, the most serious of which are renal
failure, stroke and degenerative heart disease.  Most patients did not live
much beyond 50 prior to the development of enzyme replacement therapy such
as Fabrazyme®.

The petitioners are represented by C. Allen Black, Ph.D. and Matthew
Kurzweg.  Dr. Black is a licensed patent attorney and Mr. Kurzweg is an
experienced litigator in tort matters.  Dr. Black has helped petition the
NIH for access to drug, which was denied. He is currently helping Fabry
patients petition the FDA to prevent Genzyme from sending the majority of
the drug for sale overseas.

Dr. Allen Black offered the following comments on the lawsuit:  

“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.  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 U.S. 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.  Mt. Sinai, which owns the patent
and receives millions in royalties from Genzyme, went along with the plan
and even denied full treatment to its own patients.”  

Contact information:

C. Allen Black, Ph.D. Esq.: TEL +1.412.908.3268; email:
allen at patentlawyersite.com; www.patentlawyersite.com.

Matt Kurzweg, Esq.: TEL +1.412.258.2223; email: mkurzweg at kurzweglaw.com;

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