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Home › PDUFA Calendar › RARE

RARE PDUFA Date: FAYUVI (UX111)

Page updated ·next FDA decision on the calendar ·Data as of Oct 11, 2026 9:31 PM ET
✓ Approved · the FDA decided this application on September 17, 2026, 2 days before its September 19, 2026 goal date. Decision page with the source and measured reaction.
FDA decision Approved September 17, 2026 · Ultragenyx Pharmaceutical Inc. · Sanfilippo syndrome type A (MPS IIIA)
More Rare Disease FDA decisions →All September 2026 PDUFA dates →

The story PRIOR CRL

UX111 was approved by the FDA on September 17, 2026 to treat Sanfilippo syndrome type A (MPS IIIA). Regulatory history: the FDA issued a Complete Response Letter in July 2025 - this PDUFA is the resubmission. Balance sheet: about 10 months of cash, runway ~10 mo (estimated). ELEVATED: under 12 months
Full PDUFA calendar ->Every upcoming FDA decision date, by monthNVO-sogroya . 2026-06-30Same area: Rare / Genetic . Sogroya (somapacitan)REGN-otoferlin-gene-therapy . 2026-06-30Same area: Rare / Genetic . Otoferlin gene therapy (DB-OTO)TLX . 2026-09-11Same month decision . TLX101-Px
Plain-English summary compiled by pdufa.bio from public FDA, SEC and company sources. Informational only - not investment advice.

RARE run-up into the PDUFA (last ~120 days)

$26.91$18.5
Price path to 2026-06-19. Green = local high, red = low/sell-off. Historical price action, not a forecast.

Key facts

FDA decisionApproved September 17, 2026
FDA goal date2026-09-19
Drug / candidateUX111
IndicationSanfilippo syndrome type A (MPS IIIA)
CompanyUltragenyx Pharmaceutical Inc.
Market-cap tierMid
Cohort decision-day move (history)±2% median
ClinicalTrials.govNCT02716246

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See the full 2026 PDUFA calendar →

FAQ

What did the FDA decide on RARE's UX111?
The FDA decided RARE's (Ultragenyx Pharmaceutical Inc.) application for UX111 on September 17, 2026: Approved. The FDA goal date for this application was 2026-09-19. Verify against the linked primary source.
What is RARE's drug UX111?
UX111 is Ultragenyx Pharmaceutical Inc.'s drug, approved by the FDA on September 17, 2026 for Sanfilippo syndrome type A (MPS IIIA).
What typically happens to a mid-cap stock into a PDUFA date?
Historically, mid-cap names had a median decision-day move around ±2% (763-PDUFA cohort, 2024-26). This is historical context by market-cap tier, not a prediction for this drug.
Decision-day move · mid-cap cohortn=222 · median -0.0% · 49% up
under -30%: 5 of 222 (2%)<-30-30 to -15%: 6 of 222 (3%)-15 to -7%: 7 of 222 (3%)-7 to -3%: 21 of 222 (9%)-3 to +3%: 134 of 222 (60%)±3+3 to +7%: 34 of 222 (15%)+7 to +15%: 9 of 222 (4%)+15 to +30%: 1 of 222 (0%)over +30%: 5 of 222 (2%)>+30
Distribution of next-day % moves after the FDA decision for mid-cap PDUFAs (2020–26). Red = down, green = up, blue = flat (±3%). Historical dispersion, not a forecast.