Ultragenyx lands orphan designation for 2nd drug

Ultragenyx Pharmaceutical Inc. won orphan drug designation from the Food and Drug Administration for an experimental treatment for a rare genetic metabolic disorder, called Sly syndrome.
UX-003 is the second drug from Novato-based Ultragenyx, started two years ago by BioMarin Pharmaceutical Inc. veteran Dr. Emil Kakkis, to be tagged as an orphan. The other drug, UX-001, is aimed at GNE myopathy, a severe muscle disease found in adults that weakens patients to the point of using a wheelchair.
UX-003 is designed as an enzyme replacement therapy for patients with Sly syndrome, or MPS 7, caused by a deficiency of the lysosomal enzyme Beta-glucuronidase. That enzyme is required to break down dermatan sulfate, which is found mostly in skin, and heparan sulfate, a carbohydrate expressed on the surface of all human cells.