Fabrazyme

BACKGROUND
Genzyme currently holds the exclusive manufacturing license to Fabrazyme, a life saving drug paid for by tax dollars. However, it has rationed the drug to 30%
of the recommended dose due to manufacturing errors. It does not expect to meet the current demand until late 2011 despite imposition of FDA sanctions
and oversight. As a result of the rationing, the petitioners as well as all Fabry disease victims are not receiving the prescribed dose of drug.

FDA Citizen Petition: On January 13, 2011, on behalf Joseph M. Carik and Amber Britton, who have the genetic illness Fabry disease, a petition was filed
with the FDA to allocate full doses of the drug to U.S. citizens under the FDA consent decree with Genzyme.

A copy of the citizen petition and cover letter can be downloaded at: FDA Fabrazyme petition
The January 18, 2010 press release can be downloaded at: FDA Press release

March-in Petition: Previously on August 2, 2010 on behalf Joseph M. Carik, Anita Hochendoner, and Anita Bova, a petition was filed for march-in rights with
the Secretary of the Department of Health and Human Services. The march-in petition requests that the DHHS provide a license in order to manufacture
Fabrazyme, which is used to treat Fabry disease, a genetic disorder affecting the metabolism of fats.On December 3, 2010, the NIH denied the petition. The
opinion is located here: NIH Ruling

A copy of the march-in petition and cover letter can be downloaded at: Fabrazyme petition; Cover letter
The August 2, 2010 press release can be downloaded at: Press release

March-in Rehearing: Due to Genzyme's latest manufacturing errors in its Alston Landing plant, patients will again be restricted from access to Fabrazyme.
Additionally, drug will be reallocated away from U.S. patients to overseas markets, even though overseas patients have access to the alternative treatment
Replagal, manufactured by Shire Pharmaceuticals. Genzyme originally promised the NIH that it would restore the supply in early 2011, but Genzyme has now
pushed this date back to late 2011. At this point there is no reasonable guarantee that Genzyme will ever be able to reliably supply the drug. Thus, the
petitioners request that the NIH rehear the case based on these new and disturbing developments. The Genzyme Supply Update is located here.

A copy of the march-in petition can be downloaded at: Rehearing petition
The April 5, 2011 press release can be downloaded at: Press release for rehearing

New Evidence: The petitioner have discovered that Europeans were advised to increase the dose of Fabrazyme to original level if patients did poorly on the
low-dose. American physicians were not similarly advised. Additional evidence indicates that Genzyme is providing full doses to European patients. This
information is being submitted for consideration by the FDA and NIH: Letter to NIH; Press release regarding new evidence.

Petition Support: If you or your organization would also like to support the petition splease let us know so we can provide a link and keep you informed. You
can contact me at allen@patentlawyersite.com