Full Press Release Details
Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III.
RARE
Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III
Key Takeaway: Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III
Price reaction · baseline $79.36 (2021-07-26 close) · hit after-hours · clean, no other RARE news in the window
day 0 close
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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