Strong Revenue Growth
Ultragenyx reported $139 million in revenue for Q1 2025, a 28% growth compared to Q1 2024. Crysvita contributed $103 million, marking a 25% growth over 2024, with a notable 52% growth in Latin America and Turkey.
Progress in Clinical Development
Significant progress was reported in multiple late-stage programs, including UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome. Phase 3 trials are proceeding well, with key data readouts expected in 2026.
Global Commercial Expansion
The company expanded its global footprint, particularly in Latin America with Crysvita, and Europe and Japan with Evkeeza. Successful reimbursement negotiations in Brazil and Mexico are driving growth.
Pipeline Advancements
Two BLAs are in progress, with UX111 for Sanfilippo syndrome under FDA review and DTX401 for GSDIa on track for mid-2025 submission.