Biomarin Biomarin Pharmaceutical develops enzyme therapies for serious, life-threatening diseases and conditions. We are committed to improving the lives of patients by developing novel enzyme therapies for currently unmet medical needs. BioMarin Announces FDA Approval for Naglazyme... BioMarin Announces FDA Approval for Naglazyme in the following press release. The National MPS Society is very grateful to all the families who participated in the clinical trials. These individuals were instrumental in ensuring that this treatment is now available to all individuals with MPS VI. First Specific Therapy Approved for the Treatment of MPS VI BioMarin Pharmaceutical Inc. announced today, 6/1/2005, that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Naglazyme(TM) (galsulfase), the first specific therapy approved for the treatment of mucopolysaccharidosis VI (MPS VI). As the first drug ever approved for MPS VI, Naglazyme has been granted orphan drug status in the United States, which confers seven years of market exclusivity. BioMarin plans to launch Naglazyme in the United States in approximately 30 days.Naglazyme is indicated for patients with MPS VI. Naglazyme has been shown to improve walking and stair-climbing capacity. As post-marketing clinical commitments, BioMarin has agreed with the FDA to evaluate the effect of Naglazyme treatment on skeletal dysplasia in patients under the age of 1 and to maintain a clinical surveillance program to monitor patients on commercial therapy; no extension study of Phase 3 patients was required. Clinical trials have demonstrated that Naglazyme provides clinically important benefits for MPS VI patients, specifically, improved endurance as demonstrated by the 12-minute walk test and 3-minute stair climb.Naglazyme reduced the excess carbohydrates (glycosaminoglycans, or 'GAGs') that are excreted in the urine of patients with MPS VI, an indication of enzymatic bioactivity. "I have observed the positive effect that enzyme replacement therapy with Naglazyme can have on MPS VI patients, and I am very pleased that it will soon be made commercially available to those who need it," stated Paul Harmatz, M.D., Associate Director of the Pediatric Clinical Research Center at Children's Hospital & Research Center at Oakland, California, and Principal Investigator of the Phase 3 clinical trial of Naglazyme. "With Naglazyme now approved, physicians, for the first time, have a therapeutic to treat the underlying cause of MPS VI, increasing their ability to provide better care for MPS VI patients with this life-threatening disease." "The approval of Naglazyme is a significant milestone for those whose life has been affected by MPS VI and for BioMarin," stated Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "The disease burden of MPS VI is enormous for patients, families and physicians. Naglazyme holds a very real possibility for making MPS VI a more manageable disease." Mr. Bienaime continued, "BioMarin developed Naglazyme on its own and now, with our U.S.- based sales force in place, we are ready to bring it to market. Our efforts to identify individuals with MPS VI in the years leading up to this day have positioned us to rapidly get patients on therapy come product launch. I would like to thank the individuals with MPS VI and their families and physicians as well as BioMarin employees for their years of hard work and dedication toward making Naglazyme for MPS VI a reality." An application to market Naglazyme is currently pending in the European Union. BioMarin expects to receive an opinion from the European Commission in the fourth quarter of 2005, and if positive, final approval in early 2006. BioMarin Offers Help with Insurance Questions for MPS VI BioMarin has developed a free and confidential service designed to help assist with healthcare insurance questions. The BioMarin Patient and Physician Support program (BPPS) can help families evaluate their current healthcare insurance coverage, provide information on potential healthcare insurance options that may be available in your state, and educate insurance companies about MPS VI. To contact a BPPS patient advocate, please call their toll free number: (866) 906-6100.