Genzyme's Fabrazyme at home and abroad

In my previous post, Fabry Disease Patients Have New Option, I mentioned that Shire's product, Replagal, was widely available outside the US, in competition abroad with Genzyme's product, Fabrazyme, which is the only drug approved in the US. In pursuing their various options to obtain full doses of Fabrazyme for their life-threatening disease, American patients have sued Genzyme and asked for legislation to prevent Genzyme from exporting Fabrazyme to countries where Replagal is available, while imposing rationing at home. Ed Silverman has two especially pertinent posts at his Pharmalot blog.

Genzyme Charged with Double Standard by Patients
Genzyme Charged with Double Standard, Again

Fabry disease patients have new option

Fabry disease sufferers in the US have simultaneously pursued several courses of action in hopes of obtaining relief from the life-threatening situation in which they find themselves. ABC News has a good overview. (See my earlier post below about compulsory licensing ) One hopeful outcome, the result of a letter-writing campaign by the Fabry Association, is that Shire sought and obtained approval from the European Medicines Agency to manufacture Replagal in a Lexington, MA factory. FDA approval is now being sought. The FDA previously approved a clinical trial with 140 Fabry patients who were switched from Genzyme's Fabrazyme to Shire's Replagal. The trial results were positive. FDA approval of Replagal has also been fast tracked. A review (second and third paragraphs) by Sylvie Grégoire, Shire's President of Human Genetic Therapies, presents the company's pre-approval efforts to help US patients as well as those in countries where Replagal has already been approved.

Fabry Patients Petition For Compulsory License of Fabrazyme

Fabry Patients Petition For Compulsory License of Fabrazyme

Fabry's disease is a rare, life-threatening genetic disorder for which the only FDA-approved treatment is Fabrazyme from Genzyme (now owned by Sanofi). Due to persistent and long-term production problems dating back to 2009, some Fabry patients are in declining health due to drug rationing, and others are dead.

Besides the so-called march-in petition to the National Institutes of Health filed under the auspices of the Bayh-Dole Act on April 5, 2011, an earlier petition filed in August 2010 was rejected by NIH in December 2010. Despite the strong-sounding language of the Act, no petition for a compulsory license has ever been granted.

Fabrazyme arose out of federally-funded research at Mt. Sinai Medical School, which licensed it to Genzyme. Both were sued in March 2011 by Fabry patients as another possible means to obtain relief from their disease.

There has never been a compulsory license granted in the US, but there is at least some small chance that Fabry sufferers may be the first to win one.

This post is a continuation of my previous post, Compulsory Licensing of Proprietary Drugs.

Ed Silverman has many posts on this subject at his Pharmalot blog.

Genzyme Drugs Contain Bits of Trash
Here's What the Genzyme Consent Decree Says...
Patients Sue Genzyme & Mt. Sinai Over Fabrazyme (contains link to PDF of lawsuit)
Patients Try Again to Break Genzyme Drug Patent (contains link to PDF of petition)
Genzyme Apologizes for Fabrazyme Screwup