← All organizations
Highest phase reached
Phase 4
First clinical trial
Jul 1, 2010First marketed drug
Nov 15, 2017First NDA
Nov 15, 2017Last known activity
Nov 30, 2025
Timeline
2010s
- Jul 1, 2010
Ultragenyx Inc.— Founded
- Sep 18, 2017
Dimension Therapeutics— Acquired
- Apr 17, 2018
burosumab-twza— Marketing Organization
- Apr 17, 2018
burosumab-twza— NDA Organization
2020s
- Jun 30, 2020
triheptanoin— NDA Secondary Org
- Jul 25, 2022
GeneTX Biotherapeutics, LLC— Acquired
Drugs by trial phase (25 distinct, 64 trials)
Phase 4 (2 own, 0 via acquisition)
Phase 3 (12 own, 1 via acquisition)
Phase 2 (7 own, 2 via acquisition)
Other trials (1 own, 0 via acquisition)
Indications
Approved for
Studied for
- Amyotrophic Lateral Sclerosis
- Angelman Syndrome
- Carbohydrate Metabolism, Inborn Errors
- Chronic Pain
- Disease
- Distal Myopathies
- Familial Hypophosphatemic Rickets
- Glycogen Storage Disease
- Glycogen Storage Disease Type I
- Glycogen Storage Disease Type III
- Glycogen Storage Disease Type IV
- Glycogen Storage Disease Type VII
- Hemophilia A
- Huntington Disease
- Hypophosphatemia
- Metabolic Diseases
- Motor Neuron Disease
- Movement Disorders
- Mucopolysaccharidoses
- Mucopolysaccharidosis VII
- Muscular Diseases
- Neoplasms, Connective Tissue
- Nervous System Diseases
- Nevus
- Nevus, Sebaceous of Jadassohn
- Osteomalacia
- Rett Syndrome
- Rickets
- Rickets, Hypophosphatemic
- Syndrome
Marketed drugs (3)
Subsidiaries (2 direct, 2 total across the full acquisition tree)
Recent trials (50)
- NCT03775187Lead sponsorN/A—
- NCT03773770Lead sponsorN/A—
- NCT03775174Lead sponsorN/A—
- NCT07097311CollaboratorPhase 2Nov 30, 2025
- NCT07157254Lead sponsorPhase 2Oct 13, 2025
- NCT07157254OfficialPhase 2Oct 13, 2025
- NCT06617429Lead sponsorPhase 3Dec 31, 2024
- NCT06617429OfficialPhase 3Dec 31, 2024
- NCT06636071Lead sponsorPhase 3Oct 31, 2024
- NCT06636071OfficialPhase 3Oct 31, 2024
- NCT06415344OfficialPhase 3Jul 31, 2024
- NCT06415344Lead sponsorPhase 3Jul 31, 2024
- NCT06340685CollaboratorPhase 1Jul 11, 2024
- NCT06067802CollaboratorPhase 2Dec 31, 2023
- NCT05768854Study directorPhase 3Jun 14, 2023
Details
- Founded
- Novato, CA, US Jul 1, 2010
- First NDA
- Nov 15, 2017
- Last known activity
- Nov 30, 2025
Designations held
- Orphan drug
- Fast track
- Priority review
- Breakthrough therapy