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
23
Phase 3 Programs
11
Upcoming Catalysts
3
Next Catalyst
Jan 2027
Market Overview
Stock performance and key metrics
3 upcoming, 1 past
How RARE's past catalysts played out
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
Burosumab
Hypophosphatemia
UX003
MPS 7
Bisphosphonate
Osteogenesis Imperfecta
GTX-102
Angelman Syndrome
UX111
MPS IIIA
Evinacumab
Homozygous Familial Hypercholesterolemia
GLUT1 DS
Glut1 Deficiency Syndrome
Standard of Care (SOC)
Wilson Disease
Sialic Acid Extended Release (SA-ER)
GNE Myopathy
BPS804
Hypophosphatasia
steroid regimen
GSD1
Reactive Corticosteroid Taper Regimen
Ornithine Transcarbamylase (OTC) Deficiency
SA-ER 500 mg
GNE Myopathy
UX007
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
BPS804 20mg/Kg
Osteopenia
[6,6-2H2]glucose
Glycogen Storage Disease Type IA
POST Triheptanoin
Frontal Lobe Hypometabolism
Triheptanoin oil
Huntington Disease
| Drug Name | Phase | Indication | Designations | Patent |
|---|---|---|---|---|
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 | - | - |
Burosumab | Phase 3 | Hypophosphatemia | - | - |
UX003 | Phase 3 | MPS 7 | - | - |
Bisphosphonate | Phase 3 | Osteogenesis Imperfecta | - | - |
GTX-102 | Phase 3 | Angelman Syndrome | - | - |
UX111 | Phase 3 | MPS IIIA | - | - |
Evinacumab | Phase 3 | Homozygous Familial Hypercholesterolemia | - | - |
GLUT1 DS | Phase 2 | Glut1 Deficiency Syndrome | - | - |
Standard of Care (SOC) | Phase 2 | Wilson Disease | - | - |
Sialic Acid Extended Release (SA-ER) | Phase 2 | GNE Myopathy | - | - |
BPS804 | Phase 2 | Hypophosphatasia | - | - |
steroid regimen | Phase 2 | GSD1 | - | - |
Reactive Corticosteroid Taper Regimen | Phase 2 | Ornithine Transcarbamylase (OTC) Deficiency | - | - |
SA-ER 500 mg | Phase 2 | GNE Myopathy | - | - |
UX007 | Phase 2 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | - | - |
BPS804 20mg/Kg | Phase 2 | Osteopenia | - | - |
[6,6-2H2]glucose | Phase 2 | Glycogen Storage Disease Type IA | - | - |
POST Triheptanoin | Phase 2 | Frontal Lobe Hypometabolism | - | - |
Triheptanoin oil | Phase 2 | Huntington Disease | - | - |
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 23 drug candidates in Ultragenyx Pharmaceutical Inc.'s (RARE) pipeline across Phase 3, Phase 2. Its lead program is aceneuramic acid extended-release (Ace-ER) (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 January 15, 2027. 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.