Genzyme, a Sanofi company, has begun shipping Fabrazyme (agalsidase beta) produced at its newly approved plant in Framingham, Massachusetts. As previously communicated, patients in the US are now able to return to full dosing in March. In addition, all new patients in the US are eligible to begin Fabrazyme treatment, at full dosing levels.
“The ability to meet the needs of patients in the US is an important first step in restoring unconstrained supply for all patients globally throughout the course of 2012,” said Genzyme’s president and CEO David Meeker.
In Europe the process of moving the most severely affected patients to full dose of Fabrazyme will begin in March 2012. Globally, the complete return to normal supply levels of Fabrazyme will begin in the second quarter and continue throughout the year as planned, as Genzyme works to obtain all global regulatory approvals throughout the year and to build inventory.
The Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved the manufacturing plant in Framingham, Massachusetts, for the production of Fabrazyme in January 2012.
Fabrazyme is approved for treatment of Fabry disease, an inherited condition that is characterized by excessive accumulation of the lipid GL-3 in various organs and tissues, which over time can cause renal, cardiac and cerebrovascular events. As a result, patients with Fabry disease typically have a shortened life span, and children must often cope with significant pain and disability. Fabry disease is an inherited and life threatening disease linked to the X chromosome which affects approximately 5,000 patients in the world.
Genzyme has pioneered the development and delivery of transformative therapies for patients affected by rare and debilitating diseases for over 30 years.