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Data updated: Nov 8, 2026

ULTRAGENYX PHARM INC

Biotech
Pharmaceuticals Execution: Fair

ULTRAGENYX PHARM INC is a biotechnology company with 4 FDA-approved products.

2017
Since
4
Drugs
-
Trials
2
New Drugs (2yr)
Modality:
1 Small Molecules 3 Biologics

FDA Novel Drug Designations

(2017-2020)
2 Novel 1 First-in-Class 2 Orphan 1 Priority Review 2 Fast Track
See 2 novel approvals →
2020 DOJOLVI (TRIHEPTANOIN) Orphan · Fast Track
2017 MEPSEVII (VESTRONIDASE ALFA-VJBK) First-in-Class · Orphan · Priority Review · Fast Track

ULTRAGENYX PHARM INC at a Glance

  • Fast trial execution (30 months median completion)

ULTRAGENYX PHARM INC's Key Drugs

ULTRAGENYX PHARM INC's core commercial portfolio centers on DOJOLVI, MEPSEVII, Fayuvi — its most strategically important drugs approved in the last 15 years.

DOJOLVI
Rare Disease

Long-Chain Fatty Acid Oxidation Disorders

Approved 2020
MEPSEVII
Rare Disease

Mucopolysaccharidosis VII

GUS Approved 2017
Fayuvi
Approved 2026
GENGLYCOS
Approved 2026

ULTRAGENYX PHARM INC's Recent FDA Approvals

ULTRAGENYX PHARM INC secured 2 new FDA approvals for novel drugs (NDA/BLA) in the last two years.

Fayuvi BLA
2026-09-17
rebisufligene etisparvovec-hopf
GENGLYCOS BLA
2026-08-19
pariglasgene brecaparvovec-opnr

ULTRAGENYX PHARM INC's Therapeutic Areas

Disease areas where ULTRAGENYX PHARM INC has approved drugs and an active pipeline. Weighted by commercial stage — approved drugs count most, then late-stage trials.

No indication data

ULTRAGENYX PHARM INC Pipeline Snapshot

Active clinical trials for ULTRAGENYX PHARM INC across all therapeutic areas, from ClinicalTrials.gov.

No active pipeline data available

Phase 3 Readout Calendar Pro

5 Phase 3 trials with confidence-graded primary completion dates.

Full calendar →
Q1 2027
setrusumab
Osteogenesis Imperfecta
Estimated · aging NCT06636071
Q3 2027
UX111
MPS IIIA
Estimated · aging NCT02716246
Q3 2027
Adjuvant Immunomodulatory (IM) Therapy
Mucopolysaccharidosis IIIA
Estimated · aging NCT04360265
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Anchored on CT.gov primary completion date. Topline announcements typically precede this by 3–9 months. Confidence labels combine date type (ACTUAL/ESTIMATED) with last-update freshness.

Pro Intelligence Preview

Deep insights for investors and analysts

Revenue Insights

  • • DOJOLVI leads revenue
  • • 4 key drugs tracked
View revenue breakdown →

Trial Catalysts

  • • Active trial monitoring
  • • 5 Phase 3 readouts tracked
View readout calendar →

Patent Risk

  • • Patent expiration timeline
  • • Revenue at risk analysis
View patent timeline →

Compare Companies

  • • Side-by-side pipeline analysis
  • • Revenue & market share comparison
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Execution Intelligence

  • • Phase 3: 4/13 completed
  • • Speed: 30 months avg
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Revenue forecasts • Trial milestones • Patent risk modeling

How We Calculate These Metrics

Execution Quality Score

Measures trial completion rates weighted by phase importance. Phase 3 trials count 4x more than Phase 1 because late-stage completion is harder and more valuable.

What counts as "completed": A trial that reached its planned endpoint and reported results. Trials that were terminated early, withdrawn, or suspended are not counted as completed—these often indicate safety issues, lack of efficacy, or strategic pivots.

  • Excellent (80%+): Top-tier execution, most trials reach planned endpoints
  • Good (60-79%): Strong execution with occasional early terminations
  • Fair (40-59%): Average execution, notable rate of terminated/withdrawn trials
  • Needs Improvement (under 40%): High termination rates, execution challenges