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.
4
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
16
Indications↗
Distinct diseases across all programs
43
Clinical Trials↗
All sponsored trials (all statuses)
2
Orange Book Patents
Across 1 drug with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
4 drugs in development
Approved1
Phase 31
Phase 22
Phase 12
By Therapeutic Area
top 2
Metabolic & Endocrine
350%
Neurology
117%
R&D Momentum
43/100
Pipeline Velocity
6/100
Trial Completion Rate
68/100
Pipeline Concentration
39/100
Pipeline Freshness
64/100
Strategic Signals
- ↗Active in Neurology but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Earlier
Jun 24, 2026TrialA 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
Dec 3, 2024TrialPhase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Oct 25, 2024TrialSetrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
Jul 31, 2024TrialLong-term Extension of GTX-102 in Angelman Syndrome
Jun 14, 2023TrialSetrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Feb 28, 2023TrialA Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD
Oct 18, 2022TrialClinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
4 more events
Apr 28, 2022TrialLong-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
Feb 21, 2022TrialSetrusumab vs Placebo for Osteogenesis Imperfecta
Nov 8, 2021TrialA Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Oct 18, 2021TrialSafety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
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
OtherMetabolic & EndocrineNeurologyOncologyNephrologyCardiovascular
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Metabolic & Endocrine
Neurology
Metabolic & Endocrine
Neurology
Huntington Disease
DojolviApproved
Migraine Disorder
DojolviApproved
Unknown / Other
Showing 15 of 16 programs
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)