[Ip-health] Julie Donnelly in Boston Business Journal: Genzyme patients want patent lifted

James Love james.love at keionline.org
Thu Aug 5 03:35:42 PDT 2010


Wednesday, August 4, 2010
Genzyme patients want patent lifted
Boston Business Journal - BY Julie M. Donnelly

Three patients taking Genzyme’s Fabrazyme for Fabry disease, have
petitioned the U.S. Department of Health and Human Services to use a
rarely-used legal maneuver to open up the drug to competition .On August
2, Joseph M. Carik, Anita Hochendoner, and Anita Bovaasked the federal
government to invoke so-called Bayh-Dole march-in rights and grant an
open license for other companies to use patents related to Fabrazyme.
The law is designed to protect patients when a manufacturer with a
monopoly on a drug cannot serve demand. There has been a shortage of
Fabrazyme since the temporary closure of Genzyme’s Allston plant last
summer following viral contamination.

In their petition, that patients allege that Genzyme has harmed the
public health by severely rationing the supply of agalsidase beta, the
only approved therapeutic treatment for Fabry disease.

The petition reads, in part, describing the patients’ health status:
"Their symptoms have worsened, and they are at greater risk of morbidity
and death due to complications from the disease because of the severe
and ongoing restriction in the supply of Fabrazyme. Their position is
identical to all Fabry patients because all patients are being rationed
the drug by Genzyme."

Currently, there are no other approved drugs to treat Fabry disease.
Shire plc, whose Human Genetic Therapies division is headquartered in
Massachusetts, has a drug, Replagal, which is approved in Europe and has
been submitted for approval to the U.S. Food and Drug Administration.
U.S. regulators have been making Replagal available, pre-approval, on an
emergency basis. But still, demand is not being met.

Jerry Walter, President of the National Fabry Disease Foundation, said
in an interview, "There was such a flood of patients onto the Shire
drug, through the treatment protocol, that they eventually said, ‘we
can’t take any more patients, until we find out if the drug is going to
be approved.’"

There is a third potential treatment option for Fabry patients, an oral
medication candidate produced by Amicus Therapeutics. The company is
conducting a phase 3 trial for the potential drug, and is recruiting
patients. Only patients with a certain gene mutation, and who have not
been taking Genzyme’s or Shire’s drug for at least the past six months,
are eligible for the trial. Walter said that when the shortage hit, some
patients stopped taking reduced doses of Fabrazyme to join the Amicus

James Love, Director, Knowledge Ecology International
http://www.keionline.org | http://www.twitter.com/jamie_love
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