Ultragenyx Announces Initiation of Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7

News | 12. 15. 2014

Ultragenyx Pharmaceutical

Agreement Reached With Both FDA and EMA on Pivotal Trial Design
NOVATO, Calif., Dec. 15, 2014 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the dosing of the first patient in the pivotal Phase 3 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of Mucopolysaccharidosis 7 (MPS 7, Sly syndrome).

“We are pleased to have reached alignment with both the FDA and EMA on our single pivotal study design for this devastating disease,” commented Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx. “This is an important step forward for these patients who have no approved treatment options. It is also an important milestone for Ultragenyx as it is the start of our first Phase 3 program.”

The Phase 3 global, randomized, placebo-controlled, blind-start clinical study will assess the efficacy and safety of rhGUS in 12 patients between 5 and 35 years of age. Patients will be randomized to one of four groups. One cohort begins rhGUS therapy immediately, while the other three start on placebo and cross over to rhGUS at different predefined time points in a blinded manner. This novel trial design generates treatment data from all 12 patients, improving the statistical power of the study relative to a traditional parallel-group design. Based on data from the Phase 1/2 study, patients will be dosed with 4 mg/kg of rhGUS every other week for up to a total of 48 weeks, and all groups will receive a minimum of 24 weeks of treatment with rhGUS.