The FDA approved Ultragenyx’s Fayuvi for Sanfilippo syndrome type A following a manufacturing-related delay in 2025. Children ages 2–5 who received the treatment maintained or improved their cognitive scores.
GTX-102 failed Phase 3 in Angelman syndrome. Ultragenyx will review the program and reduce expenses.
Ultragenyx’s GENGLYCOS receives accelerated FDA approval as the first gene therapy targeting the root cause of GSDIa in patients aged 8 and older.
Goldman Sachs downgraded Ultragenyx from Buy to Neutral and slashed its price target to $25 after setrusumab Phase 3 miss in osteogenesis imperfecta. GTX-102 in Angelman syndrome now key focus, but risks remain.
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.
