MYOZYME LAUNCHED WORLDWIDE
Following approval for all patients with Pompe disease in the European Union and the United States in early 2006, the launch of Myozyme occurred simultaneously in these locations and has been the fastest start for a lysosomal storage disorder (LSD) product. By year-end, the first therapy to be approved for this often deadly disease was available in 39 countries and reimbursed in 24. The expansion of global reimbursement is a priority, and eight new country reimbursements are expected in 2007. We are currently conducting a clinical study of late-onset patients and expect to have results in early 2008.
Myozyme is the fourth LSD therapy we have brought to market, all for diseases that previously had no effective treatments. In all of these diseases, early diagnosis and treatment are key for achieving optimal clinical outcomes. We have supported the development of a test to diagnose Pompe patients earlier and less invasively and have developed a newborn screening technology that we will make available to the medical community free of charge.
CEREZYME: AVAILABLE AROUND THE WORLD
More than 15 years after the initial launch of our first therapy for Gaucher disease, Cerezyme continues to grow because of its proven efficacy and safety. Cerezyme is truly a global product, available in approximately 90 countries. The long-term positive effects of Cerezyme therapy were confirmed by the results of a comprehensive study of its effects on bone health, which were published in early 2007. We continue to innovate for Gaucher patients through our phase 2 trial of a small molecule treatment and early-stage gene therapy research.
FABRAZYME: DRIVEN BY NEW DATA
We expect sales of Fabrazyme to reflect significant data from a post-marketing study published in early 2007 linking early intervention with optimal outcomes. The study indicated that observable benefits from Fabrazyme treatment are greater when patients are treated earlier in the course of the disease. These findings are consistent with data from a phase 3 clinical trial that showed long-term renal function is maintained when patients are treated before irreversible kidney damage is present. There is untapped potential to reach diagnosed Fabry patients in Europe, Latin America, and the United States who are not yet on therapy. In 2007 we will further our outreach efforts to identify new patients.
CONTINUED PROGRESS AND INVESTMENT
We continue to expand the number of patients treated with Aldurazyme, the therapy for MPS I we developed in collaboration with BioMarin Pharmaceutical Inc. Now more than 500 people in approximately 40 countries receive Aldurazyme, which was approved in Japan in late 2006.