Strong Full-Year Revenue Growth
Total revenue for 2025 was $673 million, up 20% year-over-year and exceeding the upper end of guidance.
Crysvita Franchise Performance
Crysvita generated $481 million in 2025 (North America $275M, Latin America & Turkey $177M, Europe $29M), representing 17% growth versus 2024 and exceeding guidance.
High Growth Products: Evkeeza and Dojolvi
Evkeeza revenue was $59 million, up 84% year-over-year as demand builds outside the U.S.; Dojolvi generated $96 million, up 9% year-over-year.
Global Commercial Footprint Expansion
Company is bringing treatments to patients in more than 35 countries; Evkeeza has ~350 patients across 20 countries and growing reimbursement coverage in key markets.
Robust Clinical Follow-up and Positive UX111 Data
UX111 (MPS IIIA) program now has >8 years of follow-up with sustained separation in multiple neurologic endpoints (Bayley cognition and communication) and significant, durable reductions in CSF heparan sulfate biomarkers correlated with clinical stabilization or improvement.
Near-Term Regulatory and Clinical Catalysts
Two potential approvals anticipated in 2026 (MPS IIIA/UX111 and GSD Ia/DTX401), a completed rolling BLA for DTX401 with an expected PDUFA action date in Q3 2026, and a pivotal GTX-102 (Angelman) Phase III Aspire data readout expected H2 2026.
Restructuring to Drive Path to Profitability
Strategic restructuring announced to align resources to near-term value drivers: 10% workforce reduction (~130 FTE) and guidance that combined R&D and SG&A will be flat to down low single digits in 2026 versus 2025, with combined R&D and SG&A expected to decrease at least 15% in 2027 versus 2025.
Cash and Balance Sheet Actions
As of Dec 31, 2025 the company reported $738 million in cash, cash equivalents and marketable securities and plans to consider monetizing two potential Priority Review Vouchers (PRVs) as part of its plan to reach profitability.
Concrete 2026 Revenue Guidance
2026 revenue guidance set at $730 million to $760 million (8%–13% growth over 2025), Crysvita guidance $500M–$520M, and Dojolvi guidance $100M–$110M (guidance excludes potential launch revenue).