Ultragenyx Pharmaceutical Inc.

RARE

N/A

NASDAQBiotechnology🇺🇸North America1.3K employees

Drugs in Pipeline

Phase 3 Programs

Upcoming Catalysts

Next Catalyst

Jul 15, 2026

29w

Drug Pipeline

Drug Name	Phase	Indication	Designations
aceneuramic acid extended-release (Ace-ER)	Phase 3	Hereditary Inclusion Body Myopathy	-
Bisphosphonate	Phase 3	Osteogenesis Imperfecta	-
GTX-102	Phase 3	Angelman Syndrome	-
Oral Corticosteroids	Phase 3	OTC Deficiency	-
UX003	Phase 3	MPS 7	-
UX111	Phase 3	MPS IIIA	-
Triheptanoin	Phase 3	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	-
Oral prednisolone	Phase 3	Glycogen Storage Disease Type IA	-
UX007	Phase 2	Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	-
BPS804 20mg/Kg	Phase 2	Osteopenia	-
setrusumab	Phase 2	Osteogenesis Imperfecta, Type I	-
Sialic Acid Extended Release (SA-ER)	Phase 2	GNE Myopathy	-
BPS804	Phase 2	Hypophosphatasia	-
steroid regimen	Phase 2	GSD1	-
Placebo	Phase 2	Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)	-
SA-ER 500 mg	Phase 2	GNE Myopathy	-
Reactive Corticosteroid Taper Regimen	Phase 2	Ornithine Transcarbamylase (OTC) Deficiency	-
Standard of Care (SOC)	Phase 2	Wilson Disease	-

Catalyst Timeline

1 upcoming, 0 past

⭐Phase 3Next

July 2026~GTX-102

Primary completion for GTX-102 trial (NCT06617429) in Angelman Syndrome

Regulatory Approvals

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