15-Year Anniversary and Program Approvals
Ultragenyx marked its 15-year anniversary, having taken 4 programs through approval in multiple regions and added a fifth for commercialization outside the U.S.
UX143 Progress for Osteogenesis Imperfecta
The UX143 program continues with positive safety profiles and expectations for successful Phase III study outcomes.
GTX-102 Receives Breakthrough Therapy Designation
GTX-102 for Angelman syndrome was granted Breakthrough Therapy designation by the FDA based on Phase I/II data showing developmental gains.
Strong Revenue Growth
Total revenue for the first half of 2025 grew by 20% compared to 2024, driven by double-digit growth across multiple product lines.
Successful Commercialization Efforts
The commercial team successfully launched and grew products like Crysvita, Dojolvi, and Evkeeza, with Crysvita seeing new patient starts and geographic expansion.