Ultragenyx Pharmaceutical(US:RARE)2025-05-06 20:09Pipeline & Approvals - Ultragenyx expects a PDUFA date of August 18, 2025, for UX111 in Sanfilippo syndrome (MPS IIIA) and a potential commercial launch in the second half of 2025[67, 98] - The company anticipates BLA submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa) in mid-2025, with a potential launch in 2026[78, 80, 98] - Phase 3 enrollment for GTX-102 in Angelman Syndrome (AS) is expected to be completed in the second half of 2025, with a Phase 2/3 Aurora study initiation planned for 2025[46, 49, 61, 98] - Ultragenyx plans to complete Stage 1, Cohort 4 enrollment for UX701 in Wilson Disease (WD) in the second half of 2025[88, 90, 98] - Phase 3 data readout for UX143 in Osteogenesis Imperfecta (OI) is expected in 2025[21, 28, 32] Financial Performance & Projections - Ultragenyx projects total revenue between $640 million and $670 million in 2025, representing a 14-20% annual revenue growth[94, 95] - Crysvita revenue is projected to be between $460 million and $480 million in 2025, a 12-17% increase[95] - Dojolvi revenue is expected to be between $90 million and $100 million in 2025, a 2-13% increase[95] - The company aims to achieve full-year GAAP profitability in 2027, driven by revenue growth, expense management, and monetization of Priority Review Vouchers (PRVs)[2, 96, 97, 99] - Ultragenyx reported $563 million in cash, cash equivalents, and marketable debt securities as of March 31, 2025[97]