PUBLIC COMPANY
save$RAREUltragenyx Pharmaceutical Inc.
$RARE15.22+3.19%Novato, CA, USupdated 2d ago
Ultragenyx 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.
community read0 votes
pipeline
9
marketed
3
phase 3
4
catalysts ytd
3
approvals ytd
0
recalls ytd
0
Pipeline(9)
pipeline depth9 products
4Phase 33Marketed2pre_registration
Marketed
burosumab
X-Linked Hypophosphatemic Rickets
Marketedtriheptanoin
Fatty Acid Biosynthesis Disorders
Marketedvestronidase alfa
Mucopolysaccharidosis VII (MPS VII) (Sly Syndrome )
Phase 3apazunersen sodium
Angelman Syndrome
Phase 3avalotcagene ontaparvovec
Ornithine-Transcarbamylase Deficiency
Phase 3rivunatpagene miziparvovec
Wilson Disease
Phase 3setrusumab
Osteogenesis Imperfecta
Sclerostin
pre_registrationpariglasgene brecaparvovec
Glycogen Storage Disease 1A
pre_registrationrebisufligene etisparvovec
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome )
Upcoming
Recent(90d)
nothing in the last 90 days
view all catalysts →Active trials
- phase3A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
- phase3Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
- phase3Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
- phase3Long-term Extension of GTX-102 in Angelman Syndrome
- —Mucopolysaccharidosis VII Disease Monitoring Program
- phase2A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
- —Tumor-induced Osteomalacia Disease Monitoring Program
- —Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
Insider activity
source: SEC Form 4routine RSU / option mechanics (6)
- Apr 22, 26grantHuizenga Theodore Alan · SVP, Chief Accounting Officer20,241 sh
- Apr 22, 26grantHuizenga Theodore Alan · SVP, Chief Accounting Officer7,348 sh
- Apr 16, 26grantKAKKIS EMIL D · President & CEO76,745 sh
- Apr 16, 26grantKAKKIS EMIL D · President & CEO132,693 sh
- Apr 16, 26grantHuang Dennis Karl · See Remarks23,116 sh
- Apr 16, 26grantHuang Dennis Karl · See Remarks79,936 sh
🐶 Hiring Signal
next earnings Nov 3, 2026
Where Ultragenyx Pharmaceutical Inc. is staffing up — a leading indicator of pipeline and commercial priorities.
therapeutic areaRare Disease 3
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