RESEARCH TRIANGLE PARK, N.C., Oct. 21, 2024 -- It is with deep sadness that Pappas Capital announces the passing of Dr. Ernest Mario, Senior Venture Partner and one of the founders...
FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7
News | 02. 28. 2012
Ultragenyx Pharmaceutical
February 28, 2012
Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS).
UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7. MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.
