Company

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical has 24 drugs tracked on RxWires: 1 post-market, 10 in Phase 3, 1 in Phase 2/3, 5 in Phase 2, 7 in Phase 1/2.

Pipeline

Drug	Phase	Indication
Crysvita (burosumab-twza) Treatment	Post-Market	Epidermal Nevus Syndrome
DTX401	Phase 3	Glycogen Storage Disease Type IA
UX007	Phase 3	Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
GTX-102	Phase 3	Angelman Syndrome
Triheptanoin	Phase 3	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
UX003	Phase 3	Sly Syndrome
DTX301	Phase 3	OTC Deficiency
Burosumab	Phase 3	Hypophosphatemia
Setrusumab	Phase 3	Osteogenesis Imperfecta
No Investigational Product	Phase 3	Mucopolysaccharidosis IIIA
Aceneuramic Acid Extended-Release Tablets	Phase 3	Hereditary Inclusion Body Myopathy
UX111	Phase 2/3	MPS IIIA
SA-ER 500 mg	Phase 2	GNE Myopathy
Sialic Acid Extended Release (SA-ER)	Phase 2	GNE Myopathy
BPS804 20mg/Kg	Phase 2	Osteopenia
BPS804	Phase 2	Chronic-kidney Disease Stage 5D on Stable Hemodialysis
Tridecanoic Acid	Phase 2	Rett Syndrome
scAAV8OTC	Phase 1/2	Ornithine Transcarbamylase (OTC) Deficiency
ABO-102	Phase 1/2	MPS IIIA
UX016	Phase 1/2	GNE Myopathy
[6,6-2H2]glucose	Phase 1/2	Glycogen Storage Disease Type IA
UX701	Phase 1/2	Wilson Disease
POST Triheptanoin	Phase 1/2	Frontal Lobe Hypometabolism
DTX101	Phase 1/2	Hemophilia B

Follow Ultragenyx Pharmaceutical on RxWires to get trial status changes, FDA actions, filings and patent dates as they happen.

Sourced from ClinicalTrials.gov, openFDA, SEC EDGAR and the FDA Orange Book. Not investment or medical advice.