Ultragenyx Reaffirms 2026 Guidance Amid Key Gene Therapy Milestones

Event summary

  • Q1 2026 revenue at $136M, with Crysvita contributing $93M and Dojolvi $18M.
  • Reaffirmed 2026 revenue guidance of $730M–$760M, targeting profitability in 2027.
  • FDA accepted BLAs for DTX401 (GSDIa) and UX111 (MPS IIIA) with PDUFA dates in H2 2026.
  • GTX-102 Phase 3 data for Angelman syndrome expected in H2 2026.
  • Operating expenses flat to slightly down vs. 2025, with net loss of $185M in Q1 2026.

The big picture

Ultragenyx is navigating a pivotal year with two gene therapy BLAs under review and a Phase 3 readout for Angelman syndrome. The company’s focus on cost efficiency and profitability in 2027 reflects broader industry trends toward financial discipline amid high R&D risks. Success in these programs could accelerate revenue growth, while regulatory setbacks or clinical data weaknesses pose execution risks.

What we're watching

Regulatory Approvals
Whether the FDA will approve DTX401 and UX111 by their PDUFA dates, unlocking new revenue streams.
Clinical Data
The durability of GTX-102’s efficacy in Angelman syndrome ahead of Phase 3 results.
Cost Discipline
Ultragenyx’s ability to sustain flat or declining R&D/SG&A expenses while advancing multiple pipeline candidates.