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RARE FDA Decision CRL · 2025-07-11

Updated ·next FDA decision on the calendar
The FDA's own letter · Complete Response Letter, BL 125845 (PDF), dated July 11, 2025, released under FDA's CRL transparency program.

Price into the decision (T-120)

PDUFA 7/11/25$45.78$31.0351/24/25
Daily close, T-120 (1/24/25) through T+5. Run-up high $45.78 on 2/13/25, low $31.035 on 7/10/25 (T-120 to T-1). FDA decision 7/11/25. Historical price action, not a forecast.
Positioning before the decision
On Jun 27, 2025, 14 days before the decision date, at-the-money options priced a move of ±27.6% through Jul 18, 2025 (the first listed expiry after the decision; ATM implied volatility 146%). The stock closed -4.9% at the first close across the decision and -11.7% five trading days on. A straddle priced weeks out covers ordinary movement plus the event, so this is the move the options market priced through that expiry, not the event-implied move alone. How this compares across 1,000+ decisions.

Decision facts

FDA decision date2025-07-11
DrugUX111 - (ABO-102)
IndicationSanfilippo syndrome type A (MPS IIIA)
Outcome✗ CRLprimary-sourced
Decision-day move-4.9%
T-120 run-up (low→high)+11.0% ($29.23→$41.44)
Market-cap tierMid
Cohort decision-day move (history)2.1% median (n=234)

Cohort move is the historical median absolute decision-day move for this market-cap tier across our run-up study. History, not a prediction.

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Questions

Was UX111 - (ABO-102) approved by the FDA?

Not in this review cycle. On July 11, 2025 the FDA issued a Complete Response Letter, declining to approve the application for UX111 - (ABO-102) in its current form. A CRL is not final; the primary source is linked on this page.

What was UX111 - (ABO-102) under FDA review for?

The application covered Sanfilippo syndrome type A (MPS IIIA). The FDA declined to approve the application in its current form on July 11, 2025.

How did RARE stock react to the FDA decision?

On the decision day RARE moved -4.9%. That is the recorded reaction to this specific event -- historical measurement, not a prediction, and not investment advice.