Ultragenyx
NASDAQ:RAREBiotechSan Francisco, California, United States · Est. 2014 · 550 employees
Ultragenyx Pharmaceutical is a biopharmaceutical company focused on developing novel products for serious rare and ultra-rare genetic disorders.
6
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
13
Indications↗
Distinct diseases across all programs
54
Clinical Trials↗
All sponsored trials (all statuses)
2
Orange Book Patents
Across 1 drug with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
6 drugs in development
Approved1
Phase 33
Phase 11
Preclinical3
By Therapeutic Area
top 3
Metabolic & Endocrine
225%
Neurology
113%
Dermatology
113%
R&D Momentum
49/100
Pipeline Velocity
11/100
Trial Completion Rate
68/100
Pipeline Concentration
34/100
Pipeline Freshness
59/100
Strategic Signals
- ↗Active in Metabolic & Endocrine but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Last 14 days
Sep 18, 2026
Active, not recruiting
A Randomized, Double-blind, Multicenter Study to Determine the Effect of Triheptanoin Compared With Even-chain, Medium-chain Triglycerides (MCT) on Major Clinical Events (MCEs) in Pediatric Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Sep 10, 2026
Active, not recruiting
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome
Sep 10, 2026
Active, not recruiting
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency
Sep 10, 2026
Active, not recruiting
A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome
Earlier
Sep 4, 2026TrialA Phase 1/2, First-in-human, Double-blind, Placebo-controlled Study to Assess Dose, Safety, and Efficacy of UX016 (Sialic Acid-C16 Prodrug) in Adults With GNE Myopathy
Aug 29, 2026TrialX-linked Hypophosphatemia Disease Monitoring Program (XLH-DMP)
Aug 28, 2026TrialMucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Aug 22, 2026TrialPhase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
4 more events
Aug 7, 2026TrialA Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome
Aug 1, 2026TrialExpanded Access to Triheptanoin
Jul 31, 2026TrialExpanded Access to Mepsevii
Jul 18, 2026TrialA Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)
Details
Company Info
Company Overview
$1.2B
Mkt Cap
$150.0M
Revenue
—
Cash
CEOEmil Kakkis
Founded2014
Employees550
HQSan Francisco, California, United States
ExchangeNASDAQ:RARE
Websiteultragenyx.com
LinkedInCompany Page
Therapeutic Focus
OtherNeurologyMetabolic & EndocrineOncology
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Dermatology
Metabolic & Endocrine
Neurology
Dermatology
Dermatitis, Atopic
Oral corticosteroidsPreclinical
Metabolic & Endocrine
Neurology
Huntington Disease
DojolviApproved
Migraine Disorder
DojolviApproved
Unknown / Other
Angelman Syndrome
GTX-102Phase 3
Hemiplegia
DojolviApproved
MPS IIIA
Prophylactic Immunomodulatory (IM) TherapyPreclinical
UX111Preclinical
Regulatory
Regulatory Timeline
20171 event
FDANov 15
MEPSEVII — MEPSEVII
Application BLA761047 (BLA)
20201 event
FDAJun 30
Dojolvi — DOJOLVI
Application NDA213687 (NDA)
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)