Organization (Industry)
ULTRAGENYX PHARMACEUTICAL INC.
ClinicalTrials.gov lists ULTRAGENYX PHARMACEUTICAL INC. as lead sponsor of 63 trials. It has 23 active development programs and 4 approved assets.
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Trials started per year with this organization as sponsor or collaborator, 1990 onward. The series also counts trials where the organization is a collaborator; the lead-sponsor trial count does not.
2026 is partial: 5 registered with a 2026 start date. The dashed outline is the pace from the 3 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 4.8 trial starts a year over the five years to September, under the 5 a year the method needs.
This page covers ULTRAGENYX PHARMACEUTICAL INC.: 63 lead-sponsored trials and 3 legal-entity registrations.
Disease areas (59)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Mucopolysaccharidosis VII | 8 |
| Osteogenesis Imperfecta | 7 |
| Rare Diseases | 7 |
| Intellectual Disability | 6 |
| Ornithine Carbamoyltransferase Deficiency Disease | 6 |
Showing 5 of 59 disease areas.
Trials by start year as sponsor or collaborator (17)
Shown in this order: most recent first. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Show the table of trials by start year as sponsor or collaborator
| Year | Trials started |
|---|---|
| 2026 (partial year) | 5 |
| 2025 | 2 |
| 2024 | 5 |
| 2023 | 4 |
| 2022 | 6 |
| 2021 | 8 |
| 2020 | 8 |
| 2019 | 5 |
| 2018 | 7 |
| 2017 | 6 |
Showing 10 of 17 years.
Prediction, not a registry value: median of 36 months from start to primary completion across 5 trials still running at the 2026-10-09 snapshot; registered median of 25 months across 43 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (23)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| Setrusumab | Osteogenesis Imperfecta | Phase 3 | 7 |
| GTX-102 | Angelman Syndrome | Phase 3 | 4 |
| No Investigational Product | Mucopolysaccharidosis III | Phase 3 | 1 |
| Adjuvant Immunomodulatory (IM) Therapy | Mucopolysaccharidosis III | Phase 3 | 1 |
| Oral Corticosteroids | Ornithine Carbamoyltransferase Deficiency Disease | Phase 3 | 1 |
Showing 5 of 23 active programs.
Approved assets (4)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| Triheptanoin | 7 |
| UX003 | 5 |
| Zoledronic Acid | 1 |
| Calcium/Vitamin D | 1 |
Sponsor delivery record (1)
Enrollment hit target: the share of this sponsor's lead-sponsored trials whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 37% hit target (n=30) | 44% on time (n=27) |
Journals published in (63)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Journal of inherited metabolic disease | 10 |
| Molecular genetics and metabolism | 10 |
| Neurology | 7 |
| The Journal of clinical investigation | 5 |
| Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research | 4 |
Showing 5 of 63 journals.
US granted patents (1)
Patents are not classified by disease area in the source, so this is the organization's overall portfolio, not a split by disease.
| US patents granted | Granted in the last 5 years | Most recent grant |
|---|---|---|
| 28 | 13 | 2025-12-02 |
SEC-reported annual financials (11)
Shown in this order: newest fiscal year first; SEC EDGAR XBRL companyfacts, USD as reported.
| Fiscal year | Revenue (USD) | Net income (USD) | R&D (USD) |
|---|---|---|---|
| 2025 | 673,000,000 | -575,000,000 | 750,000,000 |
| 2024 | 560,000,000 | -569,000,000 | 698,000,000 |
| 2023 | 434,000,000 | -607,000,000 | 648,000,000 |
| 2022 | 363,329,000 | -707,421,000 | 705,789,000 |
| 2021 | 351,406,000 | -454,025,000 | 497,153,000 |
| 2020 | 271,030,000 | -186,566,000 | 412,084,000 |
| 2019 | 103,714,000 | -402,727,000 | 357,355,000 |
| 2018 | 51,495,000 | -197,611,000 | 293,998,000 |
| 2017 | 2,612,000 | -302,139,000 | 231,644,000 |
| 2016 | 133,000 | -245,874,000 | 183,204,000 |
Showing 10 of 11 fiscal years.
Financing and grant events (22)
Shown in this order: most recent first.
| Date | Round type | Amount (USD) | Source |
|---|---|---|---|
| 2026-03-30 | Other financing | not disclosed | Sec extracted |
| 2025-11-03 | Royalty / licensing | 400,000,000 | Sec extracted |
| 2024-06-14 | Other financing | not disclosed | Edgar offerings |
| 2024-06-13 | Public offering | not disclosed | Sec extracted |
| 2024-06-12 | Other financing | not disclosed | Edgar offerings |
Showing 5 of 22 events.
FDA inspection record (1)
| Latest classification | Inspections |
|---|---|
| NAI | 3 |
Manufacturing and distribution sites (1)
| Facility | Location | Regulators | Member organization |
|---|---|---|---|
| Ultragenyx Pharmaceutical Inc. | Novato, US | FDA |
Climate targets, as filed (1)
| Reporting year | Net-zero target |
|---|---|
| 2025 | not disclosed |
Workforce and board diversity, as filed (1)
| Reporting year | Workforce % women | Board % women |
|---|---|---|
| 2025 | not disclosed | not disclosed |
Organization relationships (43)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| Mereo BioPharma Group PLC (United Kingdom) | Co sponsors trial with | 5 |
| Copenhagen University Hospital, Rigshospitalet | Co sponsors trial with | 2 |
| Kyowa Kirin (Japan) | Co sponsors trial with | 1 |
| Montefiore Health System | Co sponsors trial with | 1 |
| The University of Texas System | Co sponsors trial with | 1 |
Showing 5 of 43 relationships.
Latest dated events (146)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-11 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-10 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-09-19 | PDUFA decision date | Regulatory decision dates extracted from SEC filings |
| 2026-08-23 | Priority review | Regulatory decision dates extracted from SEC filings |
| 2026-08 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
Showing 5 of 146 events.
Biomarker capability evidence (272)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| ABO | Trial sponsor | Named in trial inclusion criteria |
| ACADVL | Trial sponsor | Named in trial inclusion criteria |
| ACADVL | Trial sponsor | Named in trial inclusion criteria |
| ACADVL | Trial sponsor | Named in trial inclusion criteria |
| ACADVL | Trial sponsor | Named in trial inclusion criteria |
Showing 5 of 272 evidence rows.
Not on this page
Checked, with nothing recorded for this organization: companies grouped under this page, related companies not grouped here, fda warning letters, fda untitled letters, fda recalls, fda import refusals, clia clinical laboratory certificates, and biomarkers this organization publishes on.