An end to the Farbrazyme shortage?
Sanofi's Genzyme Corp. starts shipping Fabrazyme from new plant
CAMBRIDGE, Mass.—Since 2009, when a virus was discovered in its manufacturing plant in Allston, Mass., Genzyme Corp. has take a lot of heat for shortages of the drug Fabrazyme to treat the rare genetic disorder, Fabry disease. This led to patients having to sharply reduce their dosages and even skip taking it at times. Now, however, there may be a light at the end of the tunnel, as the company, now owned by Sanofi, announced March 2 that it hasbegun shipping Fabrazyme produced at its newlyapproved plant in Framingham, Mass.
According to Genzyme,all patients in the United States can return to full dosing this month. Inaddition, all newly diagnosed patients in the United States are eligible to begin Fabrazymetreatment at full dosing levels.
"The ability to meet the needs of patients in the U.S. is an importantfirst step in restoring unconstrained supply for all patients globallythroughout the course of 2012," said Genzyme's president and CEO DavidMeeker.
In Europe, the process of moving the most severely affected patients tofull doses of Fabrazyme will begin this month. Globally, the completereturn to normal supply levels of Fabrazyme will begin in the secondquarter and continue throughout the year, as Genzyme works toobtain all global regulatory approvals throughout the year and to buildinventory.
The Food and Drug Administration (FDA) and the European Medicines Agency(EMA) approved the manufacturing plant in Framingham, Mass., for theproduction of Fabrazyme in January 2012.
Fabrazyme is approved for treatment of Fabry disease, an inheritedcondition that is characterized by excessive accumulation of the lipidGL-3 in various organs and tissues, which over time can cause renal,cardiac and cerebrovascular events. As a result, patients with Fabrydisease typically have a shortened life span, and children must oftencope with significant pain and disability. Fabry disease is an inheritedand life threatening disease linked to the X chromosome which affectsapproximately 5,000 patients in the world.
