BioMarin Pharmaceutical Inc. has announced that the FDA has granted marketing approval for Naglazyme™ (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.”
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.