Full Press Release Details
Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA).
RARE
Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Key Takeaway: Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Price reaction · baseline $42.49 (2025-02-14 close) · hit after-hours · 1 other RARE headline(s) in the window, move may be shared
day 0 close · peak
+2.7%
BiopharmaWatch Analysis
From our catalyst data and publicly available data · not financial adviceBest trade, last catalyst
+43%
120-day peak, hindsight
Typical move
2.9%
average across 6 past catalysts
Cash runway
~13 mo
Low dilution risk
Lead asset
BPS804
Phase 2 · Hypophosphatasia