NOVATO, Calif., Oct. 07, 2024 (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 that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type I, III, or IV in patients 2 years of age and older.

'FDA designation of setrusumab as a Breakthrough Therapy emphasizes the seriousness of osteogenesis imperfecta and the impact of this disease on people and their families affected by this disorder,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. 'The designation is also recognition of the significant clinical benefit observed in the Phase 2 portion of the Orbit study and supports our work to expeditiously bring this investigational therapy to patients who currently have no approved treatment option.”