Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be presented at WORLDSymposium™ 2026 "Today, with no approved treatment options to address the relentless progression of Sanfilippo syndrome type A, families must watch helplessly as their children lose the ability to communicate, play, move, and even eat before ultimately succumbing to this devastating and fatal disea ...

Core Insights - Ultragenyx Pharmaceutical Inc. reported preliminary total revenue for 2025 of $672 million to $674 million, exceeding the top end of guidance and representing approximately 20% growth compared to 2024 [1][3] - The company anticipates significant catalysts in 2026, including two potential gene therapy approvals and pivotal Phase 3 data for GTX-102 in Angelman syndrome [1][2] Financial Update - Preliminary revenue for 2025 is estimated at $672 million to $674 million, with Crysvita revenue projected at $480 million to $482 million, reflecting approximately 17% growth from 2024 [3] - Dojolvi revenue for 2025 is estimated at $95 million to $97 million, indicating approximately 9% growth compared to 2024 [3] - As of December 31, 2025, the company reported cash and investments of approximately $735 million [4] Clinical and Regulatory Catalysts - The company plans to present data from the Phase 3 Orbit and Cosmic studies of UX143 (setrusumab) at the J.P. Morgan Healthcare Conference on January 12, 2026 [7] - The rolling submission for DTX401 (pariglasgene brecaparvovec) for glycogen storage disease type Ia was completed in December 2025, with a PDUFA date anticipated in Q3 2026 [7] - UX111 (rebisufligene etisparvovec) for Sanfilippo syndrome type A is on track for BLA resubmission in early 2026 [7] - GTX-102 (apazunersen) data from the Phase 3 Aspire study is expected in the second half of 2026 [7] - Enrollment for UX701 (rivunatpagene miziparvovec) in the pivotal Cyprus2+ study is complete, with data expected in the first half of 2026 [7] Company Overview - Ultragenyx is focused on developing and commercializing therapies for serious rare and ultra-rare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [9] - The management team has extensive experience in the development and commercialization of rare disease therapeutics, emphasizing efficient drug development [10]