BUZZ-Ultragenyx Pharma rises as FDA accepts genetic disorder therapy for review

Drugmaker Ultragenyx Pharmaceutical's RARE.O shares rise 2.1% to $43.37 in morning trade

Co says the U.S. FDA has accepted for review its marketing application for its experimental gene therapy to treat a rare pediatric genetic disease

Co is seeking accelerated approval for its therapy, UX111, as a treatment for Sanfilippo syndrome, a disorder that primarily affects the brain and spinal cord

Co expects a regulatory decision by August 18, 2025

Ultragenyx's marketing application includes long-term data from studies in which patients treated with the therapy showed improvement in scores of a commonly used evaluation scale that measures cognition, mobility and behavior in patients, the co said

RARE has fallen ~7% in the last 12 months