Ultragenyx reports Q1 2026 revenue of $136M, reaffirms 2026 guidance, and expects Phase 3 data for Angelman syndrome later this year.

Ultragenyx Pharmaceutical reported $136 million in total revenue for Q1 2026, with key products Crysvita and Dojolvi contributing $93 million and $18 million respectively. The company reaffirmed its 2026 revenue guidance of $730 million to $760 million and expects combined R&D and SG&A expenses to remain flat or slightly decrease compared to 2025. Ultragenyx highlighted promising long-term Phase 1/2 data for GTX-102 in treating Angelman syndrome, with Phase 3 results expected in the second half of 2026. The company is also progressing with regulatory reviews for gene therapies DTX401 and UX111, targeting approvals later this year, and aims for profitability in 2027.