FDA decision (PDUFA) — RARE (Ultragenyx) UX111

Resolution

Frozen rule: 'FDA's own action on the UX111 marketing application: the approval letter or the Drugs@FDA entry recording the action, or Abeona's 8-K disclosing the agency's decision. A CRL or a delay past the date is a NO.' — OUTCOME: FDA approved UX111 (Fayuvi) 2026-09-17, two days ahead of the 9/19 PDUFA date; 'on/around' satisfied. Full approval, first treatment for Sanfilippo A. Correction for the record: call text said (ABEO); UX111 is Ultragenyx (RARE). [2026-10-18 eric-stamp QAF-2] Labelled no-direction. EHIQ was 50% at open (ehiq_prob_at_open 0.50, the auto-sourced placeholder published 2026-08-06) and 75% final (ehiq_prob 0.75, calibrated 2026-09-09). The headline grades the at-open value; 0.50 has no direction, so this call is neither a hit nor a miss. The 0.75 is the final value (SCORING_POLICY §3 secondary track) and is not the graded probability. The stored hit column, actual_outcome and every probability are unchanged; ties stay in the hit-rate denominator (SCORING_POLICY §5). [2026-10-18 eric-stamp QAU-D4] Outcome recorded: YES. FDA press announcement, For Immediate Release 2026-09-17 (https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type): "Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A." Approved two days before the 2026-09-19 PDUFA date. EHIQ was 50% at open (75% final), so the verdict stays NO DIRECTION (neither a hit nor a miss); recording the outcome adds the row to Brier at 0.25. hit, resolution, resolved_at and every probability are unchanged.