Ultragenyx’s one-time gene therapy DTX401 just got FDA Priority Review for Glycogen Storage Disease Type Ia (GSDIa). Possible approval by August 23, 2026 – the first treatment that could fix the root cause instead of just managing symptoms.
Analysts react to disappointing ORBIT and COSMIC trial results for Ultragenyx and Mereo BioPharma, highlighting risks, ratings changes, and what could come next.
Ultragenyx's setrusumab fails to reduce fractures in osteogenesis imperfecta patients in Phase 3 trials, though bone density improved. Company plans expense cuts.
JPMorgan is optimistic about Ultragenyx's drug setrusumab (UX143) and expects big gains ahead of key study updates.
