Literally a lifeline for babies with Pompe disease, Myozyme - the first therapy for this rare and often fatal disorder - is nearing launch in the second quarter of 2006.

Our massive effort to bring an effective therapy for Pompe disease to market achieved success in January 2006, when European regulators issued a positive opinion on Myozyme. We anticipate approval in the United States in the spring and are preparing for commercial launch in the second quarter. Later in 2006, we plan to file for approval in Japan. Myozyme has orphan drug designation in all of these jurisdictions.

The challenge of Pompe disease
Myozyme will be the first treatment approved for Pompe disease, a debilitating and often fatal muscle disorder resulting from an inherited enzyme deficiency. We focused our first efforts on the infantile-onset form of the disease because many of these patients die before reaching their first birthday. In our pivotal clinical study of this population, all patients who began receiving Myozyme by 6 months of age were alive at 18 months, and 83 percent were free of invasive ventilator support. In fall 2005, we began a trial of late-onset patients, who may develop symptoms during childhood or adulthood. Late-onset patients often require breathing assistance and mobility aids, and their lifespan is shortened. We are also conducting a small-scale study of late-onset patients age 5 to 15. These clinical investments will continue post-launch as we seek detailed knowledge of Pompe disease in all patient populations.

Ongoing commitment
Developing and commercializing a treatment for Pompe disease has required an immense investment that would not have been possible for us a decade ago. Myozyme represents the best of four different approaches we studied to meet the many challenges posed by Pompe disease. We built on our experience base of successful treatments for three other enzyme deficiencies, searched the world for patients, and even developed a technology for newborn screening that we will make available at no cost to the medical community.

As part of creating a system to provide optimal patient care, we strive to create awareness among physicians and patients. While we have been able to work with existing centers of excellence for genetic diseases, we have also reached out to a new category of specialists in neuromuscular medicine because of the particular characteristics of Pompe disease. We estimate that Pompe disease affects fewer than 10,000 patients globally; however, the therapy requires more than 20 times as much protein product as our other enzyme replacement therapies. Accordingly, we have made significant manufacturing investments well in advance of regulatory approvals in order to fulfill the needs of clinical trials and to ensure a consistent capacity after launch. Prior to approval, we have been supplying more than 250 patients with Myozyme through clinical trials and expanded access programs.