COMPANY
Ultragenyx Pharmaceutical
FDA-approved therapeutic company Clinical Phase 3
Timeline
- Trial start —: MPS IIIA Disease Monitoring Program source ↗
- Trial start Phase 1/Phase 2: First-in-human Study of UX016 in GNEM source ↗
- FDA FDA New drug/biologic (novel): Fayuvi (rebisufligene etisparvovec-hopf) — BLA125845, Sep 17, 2026. Applicant at approval: Ultragenyx Pharmaceutical Inc. source ↗
- FDA FDA New drug/biologic (novel): GENGLYCOS (pariglasgene brecaparvovec-opnr) — BLA125858, Aug 19, 2026. Applicant at approval: Ultragenyx Pharmaceutical, Inc. source ↗
- Trial start Phase 2: A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) source ↗
- Trial start 3: Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) source ↗
- Trial start —: Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program source ↗
- Trial start Phase 3: Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta source ↗
- Trial start Phase 3: Long-term Extension of GTX-102 in Angelman Syndrome source ↗
- Trial start Phase 3: Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta source ↗
- Trial start Phase 3: A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD source ↗
- Trial start Phase 3: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency source ↗
- Trial start —: Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program source ↗
- Trial start —: Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III source ↗
- Trial start Phase 2: Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta source ↗
- Trial start Phase 2/Phase 3: Setrusumab vs Placebo for Osteogenesis Imperfecta source ↗
- Trial start —: Tumor-induced Osteomalacia Disease Monitoring Program source ↗
- Trial start —: Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program source ↗
- Trial start Phase 3: A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) source ↗
- Trial start Phase 1/Phase 2: Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) source ↗
- Trial start 1: A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease source ↗
- Trial start —: Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease source ↗
- Trial start —: Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease source ↗
- Trial start —: Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III) source ↗
- Trial start —: Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa source ↗
Upcoming milestones
- 2027-01 (ESTIMATED) · Phase 3 · Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta · NCT06636071
- 2027-08 (ESTIMATED) · Phase 3 · A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD · NCT05933200
- 2027-08 (ESTIMATED) · Phase 3 · Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA · NCT04360265
- 2027-09 (ESTIMATED) · Phase 3 · Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency · NCT05345171
- 2028-12 (ESTIMATED) · Phase 1/Phase 2 · First-in-human Study of UX016 in GNEM · NCT07511556
- 2029-02 (ESTIMATED) · Phase 3 · Long-term Extension of GTX-102 in Angelman Syndrome · NCT06415344
- 2029-03 (ESTIMATED) · 1 · A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease · NCT04884815
- 2029-03 (ESTIMATED) · I · Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH · NCT02716246
Clinical trials (lead sponsor) Showing 50 of 63
| Record | Title | Phase | Status | Start | Primary completion |
|---|---|---|---|---|---|
| NCT05125809 | Setrusumab vs Placebo for Osteogenesis Imperfecta | Phase 2/Phase 3 | Active, not recruiting | Feb 21, 2022 | Oct 20, 2025 (actual) |
| NCT07855562 | MPS IIIA Disease Monitoring Program | Not listed | Not yet recruiting | 2026-11 | 2038-10 (estimated) |
| NCT06636071 | Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta | Phase 3 | Active, not recruiting | Oct 25, 2024 | 2027-01 (estimated) |
| NCT04884815 | A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease | 1 | Active, not recruiting | Sep 27, 2021 | 2029-03 (estimated) |
| NCT02716246 | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | I | Recruiting | Apr 25, 2016 | 2029-03 (estimated) |
| NCT05933200 | A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD | Phase 3 | Active, not recruiting | Feb 28, 2023 | 2027-08 (estimated) |
| NCT06415344 | Long-term Extension of GTX-102 in Angelman Syndrome | Phase 3 | Active, not recruiting | Jul 31, 2024 | 2029-02 (estimated) |
| NCT07157254 | A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) | Phase 2 | Active, not recruiting | Oct 13, 2025 | 2030-01 (estimated) |
| NCT05345171 | Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency | Phase 3 | Active, not recruiting | Oct 18, 2022 | 2027-09 (estimated) |
| NCT07511556 | First-in-human Study of UX016 in GNEM | Phase 1/Phase 2 | Recruiting | 2026-10 | 2028-12 (estimated) |
| NCT03651505 | X-linked Hypophosphatemia Disease Monitoring Program | Not listed | Active, not recruiting | Jul 16, 2018 | 2032-12 (estimated) |
| NCT03604835 | Mucopolysaccharidosis VII Disease Monitoring Program | Not listed | Recruiting | Jan 29, 2018 | 2032-04 (estimated) |
| NCT06636383 | Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program | Not listed | Recruiting | Nov 4, 2024 | 2036-12 (estimated) |
| NCT06617429 | Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) | 3 | Active, not recruiting | Dec 3, 2024 | Jul 10, 2026 (actual) |
| NCT03773770 | Expanded Access to Triheptanoin | Not listed | Available | Not listed | Not listed |
| NCT03775174 | Expanded Access to Mepsevii | Not listed | Available | Not listed | Not listed |
| NCT04360265 | Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA | Phase 3 | Enrolling by invitation | Sep 28, 2020 | 2027-08 (estimated) |
| NCT04783428 | Tumor-induced Osteomalacia Disease Monitoring Program | Not listed | Active, not recruiting | Jan 31, 2022 | Feb 28, 2032 (estimated) |
| NCT04632953 | Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program | Not listed | Active, not recruiting | Nov 30, 2021 | 2035-12 (estimated) |
| NCT03636438 | Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency | Not listed | Active, not recruiting | Aug 30, 2018 | 2029-12 (estimated) |
| NCT05139316 | A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) | Phase 3 | Completed | Nov 8, 2021 | Feb 20, 2024 (actual) |
| NCT03970278 | Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia | Not listed | Completed | Jul 15, 2019 | Feb 25, 2025 (actual) |
| NCT05768854 | Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta | Phase 3 | Active, not recruiting | Jun 14, 2023 | Oct 23, 2025 (actual) |
| NCT04259281 | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome | Phase 1/Phase 2 | Completed | Feb 24, 2020 | Jan 8, 2025 (actual) |
| NCT05312697 | Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta | Phase 2 | Terminated | Apr 28, 2022 | Jul 7, 2022 (actual) |
| NCT04708015 | Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa | Not listed | Completed | Nov 5, 2020 | Mar 4, 2022 (actual) |
| NCT04990388 | Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) | Phase 1/Phase 2 | Terminated | Oct 18, 2021 | Mar 20, 2023 (actual) |
| NCT05196165 | Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III | Not listed | Terminated | May 20, 2022 | Mar 2, 2023 (actual) |
| NCT02214160 | Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies | Phase 2 | Completed | Dec 9, 2014 | Dec 3, 2020 (actual) |
| NCT04088734 | Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | Phase 1/Phase 2 | Terminated | Sep 18, 2019 | Mar 10, 2022 (actual) |
| NCT03118570 | A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 | Phase 2 | Completed | Sep 11, 2017 | Oct 1, 2019 (actual) |
| NCT03216486 | An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis Imperfecta | Phase 2 | Withdrawn | Oct 31, 2017 | Nov 1, 2018 (estimated) |
| NCT04909346 | Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease | Not listed | Terminated | Jun 23, 2021 | Nov 17, 2022 (actual) |
| NCT02736188 | Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) | Phase 3 | Terminated | May 2, 2016 | Jan 10, 2018 (actual) |
| NCT02991144 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency | Phase 1/Phase 2 | Completed | Jul 31, 2017 | Dec 16, 2021 (actual) |
| NCT04812106 | Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program | Not listed | Terminated | Jul 25, 2022 | Oct 27, 2022 (actual) |
| NCT03517085 | Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa) | Phase 1/Phase 2 | Completed | May 18, 2018 | Nov 2, 2021 (actual) |
| NCT02931682 | Observational Study of Males With Creatine Transporter Deficiency | Not listed | Terminated | 2016-12 | Oct 24, 2022 (actual) |
| NCT01406977 | Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP) | Phase 2 | Completed | 2011-07 | 2012-09 (actual) |
| NCT01406548 | Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral Density | Phase 2 | Completed | 2011-07 | 2013-10 (actual) |
| NCT01806610 | Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic Kidney Disease | Phase 2 | Withdrawn | 2013-08 | 2014-04 (estimated) |
| NCT04574830 | Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III) | Not listed | Completed | Nov 23, 2020 | Jun 30, 2022 (actual) |
| NCT04531189 | Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease | Not listed | Completed | Dec 11, 2020 | Mar 25, 2022 (actual) |
| NCT04717453 | Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency | Not listed | Terminated | Oct 6, 2020 | Dec 15, 2021 (actual) |
| NCT02971969 | Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B | Not listed | Completed | 2017-01 | Nov 6, 2021 (actual) |
| NCT01417091 | Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta | Phase 2 | Completed | 2011-06 | 2012-12 (actual) |
| NCT01886378 | A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) | Phase 2 | Completed | Feb 6, 2014 | Aug 25, 2016 (actual) |
| NCT03768817 | Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program | Not listed | Completed | Jan 30, 2019 | Jun 8, 2020 (actual) |
| NCT02432144 | A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) | Phase 3 | Completed | Nov 10, 2015 | Jan 14, 2019 (actual) |
| NCT02230566 | A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) | 3 | Completed | 2014-12 | 2016-05 (actual) |
FDA records (last 2 years)
- FDA New drug/biologic (novel): Fayuvi (rebisufligene etisparvovec-hopf) — BLA125845, Sep 17, 2026. Applicant at approval: Ultragenyx Pharmaceutical Inc. source ↗
- FDA New drug/biologic (novel): GENGLYCOS (pariglasgene brecaparvovec-opnr) — BLA125858, Aug 19, 2026. Applicant at approval: Ultragenyx Pharmaceutical, Inc. source ↗
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