Full Press Release Details
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA).
RARE
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Key Takeaway: Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Price reaction · baseline $31.035 (2025-07-10 close) · hit after-hours · clean, no other RARE news in the window
day 0 close · peak
-4.9%
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