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
4
Next Catalyst
This month
Market Overview
Stock performance and key metrics
4 upcoming, 1 past
GTX-102 Phase 3 Est. Readout
Primary completion for GTX-102 trial (NCT06617429) in Angelman Syndrome
Source3 more catalysts beyond 14 days
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How RARE's past catalysts played out
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 | Patent |
|---|---|---|---|---|
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 | - | - |
GNE Myopathy
2 drugs in this indication
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
2 drugs in this indication
MPS IIIA
1 drug in this indication
Hypophosphatasia
1 drug in this indication
GSD1
1 drug in this indication
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
1 drug in this indication
OTC Deficiency
1 drug in this indication
Hereditary Inclusion Body Myopathy
1 drug in this indication
MPS 7
1 drug in this indication
Ornithine Transcarbamylase (OTC) Deficiency
1 drug in this indication
Angelman Syndrome
1 drug in this indication
Wilson Disease
1 drug in this indication
Osteopenia
1 drug in this indication
Osteogenesis Imperfecta, Type I
1 drug in this indication
Osteogenesis Imperfecta
1 drug in this indication
Glycogen Storage Disease Type IA
1 drug in this indication
Source: CatalystAlert database (ClinicalTrials.gov, SEC EDGAR)
Full breakdown — the signals behind the verdict
Frequently asked questions
What is in Ultragenyx Pharmaceutical Inc.'s drug pipeline?→
CatalystAlert tracks 17 drug candidates in Ultragenyx Pharmaceutical Inc.'s (RARE) pipeline across Phase 3, Phase 2. Its lead program is Bisphosphonate (Phase 3). Each drug's phase, indication and upcoming catalysts are listed on this page.
When is Ultragenyx Pharmaceutical Inc.'s next FDA catalyst?→
Ultragenyx Pharmaceutical Inc.'s (RARE) next tracked catalyst is a Phase 3 expected July 15, 2026. See the catalyst timeline on this page for the full schedule of PDUFA dates, trial readouts and FDA events.
How do I track RARE biotech catalysts?→
Add RARE to your CatalystAlert watchlist for alerts ahead of each catalyst, and follow Ultragenyx Pharmaceutical Inc.'s live catalyst calendar, drug pipeline and clinical-trial timeline on this page — sourced from ClinicalTrials.gov, FDA and SEC filings.