On Nov. 15, 2017, Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration has approved MEPSEVII™ (vestronidase alfa), the first medicine approved for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII, Sly syndrome). MEPSEVII™ is an enzyme replacement therapy designed to replace the deficient lysosomal enzyme beta-glucuronidase in MPS VII patients. The effect of MEPSEVII™ on the central nervous system manifestations of MPS VII has not been determined.
MEPSEVII™ was evaluated by the FDA with Priority Review, which is reserved for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. Designation of a drug as “Priority” does not alter the scientific/medical standard for approval or the quality of evidence necessary. In Europe, the European Medicines Agency (EMA) is currently reviewing the Marketing Authorization Application (MAA) for vestronidase alfa, and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the first half of 2018.
“I am thrilled beyond belief to see this treatment advance after more than 40 years of work and anticipation. Thanks to Ultragenyx for making it happen,” said William S. Sly, Chairman Emeritus, Department of Biochemistry at St. Louis University, who first identified and characterized MPS VII. “I hope that this treatment will follow the other successful examples of enzyme therapy for LSDs and help improve the lives of patients with this rare disease.” Former 2016-2017 Lysosomal Disease Network fellow Michael Flanagan, PhD, had the privilege of being partly mentored by Dr. Sly during his LDN fellowship at St. Louis University.