Full Press Release Details
Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA).
RARE
Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Key Takeaway: Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Price reaction · baseline $21.42 (2026-04-01 close) · hit after-hours · 2 other RARE headline(s) in the window, move may be shared
day 0 close · peak
+4.8%
BiopharmaWatch Analysis
From our catalyst data and publicly available data · not financial adviceBest trade, last catalyst
+20%
120-day peak, hindsight
Typical move
3.5%
average across 15 past catalysts
Cash runway
~12 mo
Medium dilution risk
Lead asset
BPS804
Phase 2 · Osteopenia