一次情報の記録
重要申請受理
Ultragenyx、UX111 BLA再提出に対しIRLを受領
同社は2026年1月に再提出したUX111のBLAに対し、不完全回答書（Incomplete Response Letter）を受領し、追加のCMC資料を求められた。同社は、求められた情報をさらに再提出する予定である。
主要事実
出典の抜粋
EX-99.1 2 rare-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Media Jess Rowlands media@ultragenyx.com Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update 2025 total revenue of $673 million, Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million 2026 total revenue from current products expected to be between $730 million to $760 million Initiated a strategic restructuring plan to significantly reduce and focus expenses and headcount, reiterate path to profitability in 2027 2026 catalysts include two potential approvals and expected pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif. – February 12, 2025 – Ultrag