Ultragenyx Pharmaceutical Inc.
RAREUltragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.
Drugs in Pipeline
17
Phase 3 Programs
9
Upcoming Catalysts
3
Next Catalyst
May 22, 2026
14wMarket Overview
Stock performance and market intelligence
3 upcoming, 1 past
FDA PDUFA Date DTX401 (standard)
For glycogen storage disease type Ia (GSDIa). BLA filing. Extracted from SEC filing: 8-K
SourceGTX-102 Phase 3 Results Expected
Primary completion for GTX-102 trial (NCT06617429) in Angelman Syndrome
Sourcesetrusumab Phase 3 Results Expected
Primary completion for setrusumab trial (NCT06636071) in Osteogenesis Imperfecta
SourceAI Predictions & Institutional Flow
Machine learning insights and smart money movement
FDA Submissions
Pending NDA/BLA submissions with predicted PDUFA dates
Drug Pipeline
Clinical development programs and drug candidates
Bisphosphonate
Osteogenesis Imperfecta
GTX-102
Angelman Syndrome
UX111
MPS IIIA
aceneuramic acid extended-release (Ace-ER)
Hereditary Inclusion Body Myopathy
Oral Corticosteroids
OTC Deficiency
Triheptanoin
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Oral prednisolone
Glycogen Storage Disease Type IA
setrusumab
Osteogenesis Imperfecta, Type I
UX003
MPS 7
Reactive Corticosteroid Taper Regimen
Ornithine Transcarbamylase (OTC) Deficiency
Sialic Acid Extended Release (SA-ER)
GNE Myopathy
BPS804
Hypophosphatasia
steroid regimen
GSD1
SA-ER 500 mg
GNE Myopathy
Standard of Care (SOC)
Wilson Disease
UX007
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
BPS804 20mg/Kg
Osteopenia
| Drug Name | Phase | Indication | Designations |
|---|---|---|---|
Bisphosphonate | Phase 3 | Osteogenesis Imperfecta | - |
GTX-102 | Phase 3 | Angelman Syndrome | - |
UX111 | Phase 3 | MPS IIIA | - |
aceneuramic acid extended-release (Ace-ER) | Phase 3 | Hereditary Inclusion Body Myopathy | - |
Oral Corticosteroids | Phase 3 | OTC Deficiency | - |
Triheptanoin | Phase 3 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | - |
Oral prednisolone | Phase 3 | Glycogen Storage Disease Type IA | - |
setrusumab | Phase 3 | Osteogenesis Imperfecta, Type I | - |
UX003 | Phase 3 | MPS 7 | - |
Reactive Corticosteroid Taper Regimen | Phase 2 | Ornithine Transcarbamylase (OTC) Deficiency | - |
Sialic Acid Extended Release (SA-ER) | Phase 2 | GNE Myopathy | - |
BPS804 | Phase 2 | Hypophosphatasia | - |
steroid regimen | Phase 2 | GSD1 | - |
SA-ER 500 mg | Phase 2 | GNE Myopathy | - |
Standard of Care (SOC) | Phase 2 | Wilson Disease | - |
UX007 | Phase 2 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | - |
BPS804 20mg/Kg | Phase 2 | Osteopenia | - |
Regulatory & News
Approvals, filings, and latest developments