Ultragenyx Pharma rises as FDA accepts genetic disorder therapy for review

Ultragenyx Pharmaceutical

18/02/2025 16:10

** 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

 (Reporting by Siddhi Mahatole)
 ((Siddhiprabhanjan.mahatole@thomsonreuters.com))