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Home › PDUFA Calendar › RARE

RARE PDUFA Date: DTX401 AAV gene therapy (pariglasgene brecaparvovec)

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 August 19, 2026, 4 days before its August 23, 2026 goal date. Decision page with the source and measured reaction.
FDA decision Approved August 19, 2026 · Ultragenyx Pharmaceutical Inc. · Glycogen Storage Disease Type Ia (GSDIa)
More Obesity & Metabolic FDA decisions →All August 2026 PDUFA dates →

The story

DTX401 AAV gene therapy (pariglasgene brecaparvovec) was approved by the FDA on August 19, 2026 to treat Glycogen Storage Disease Type Ia (GSDIa). 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)MRNA . 2026-08-05Same month decision . mRNA-1010
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 August 19, 2026
FDA goal date2026-08-23
Drug / candidateDTX401 AAV gene therapy (pariglasgene brecaparvovec)
IndicationGlycogen Storage Disease Type Ia (GSDIa)
CompanyUltragenyx Pharmaceutical Inc.
Market-cap tierMid
Cohort decision-day move (history)±2% median
ClinicalTrials.govNCT03517085 · completed

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See the full 2026 PDUFA calendar →

FAQ

What did the FDA decide on RARE's DTX401 AAV gene therapy (pariglasgene brecaparvovec)?
The FDA decided RARE's (Ultragenyx Pharmaceutical Inc.) application for DTX401 AAV gene therapy (pariglasgene brecaparvovec) on August 19, 2026: Approved. The FDA goal date for this application was 2026-08-23. Verify against the linked primary source.
What is RARE's drug DTX401 AAV gene therapy (pariglasgene brecaparvovec)?
DTX401 AAV gene therapy (pariglasgene brecaparvovec) is Ultragenyx Pharmaceutical Inc.'s drug, approved by the FDA on August 19, 2026 for Glycogen Storage Disease Type Ia (GSDIa).
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.