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Ultragenyx Receives Approval of Clinical Trial Application (CTA)
for a Phase 1/2 Trial TestingUX003 in Mucopolysaccharidosis Type 7 (MPS 7)

NOVATO, CA – August 14, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company, received Clinical Trial Application (CTA) approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. to conduct a Phase 1/2 clinical trial of UX003, recombinant human β-glucuronidase, in MPS 7, a rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme β-glucuronidase. MPS 7 is a severe multi-system disease resulting in cellular and organ dysfunction. There is no approved drug therapy.