Company intelligence / Profile preview
Ultragenyx Pharmaceutical Inc.
Novato, California, United Statesultragenyx.com ↗
- Ownership
- Public · RARE
- Employees
- ~651
- Development stage
- Other
01
Overview
Ultragenyx has four approved products: Crysvita (burosumab) for XLH and tumor-induced osteomalacia, Mepsevii (vestronidase alfa) for MPS VII, Dojolvi (triheptanoin) for LC-FAOD, and Evkeeza (evinacumab-dgnb) for HoFH. The company reported preliminary 2024 total revenue of $555-560 million and expects 2025 revenue of $640-670 million. Key late-stage programs include UX143 (setrusumab) for osteogenesis imperfecta with a Phase 3 second interim analysis expected mid-2025, and GTX-102 for Angelman syndrome with Phase 3 enrollment expected to complete in second half of 2025.[7][9]
- Therapeutic areas
- Metabolic DisordersEndocrinologyRare DiseasesCardiovascularNeurology
- Modalities
- Biologicssmall moleculesgene therapiesantisense oligonucleotides (ASO)mRNA therapeutics[7]
- Industry
- Biotech
02
Drug pipeline
20 assets19 more assets in the full pipeline.
03
Partnerships
GeneTxKyowa Hakko KirinMereo BiopharmaDaiichi SankyoRegeneron[7]
04
Financials
- Market cap
- $1.5B
- Total revenue
- $673M
- Enterprise value
- $2.5B
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