原始出處紀錄
重大申請已受理
Ultragenyx：FDA 受理 UX111 BLA，設定 PDUFA 日期為 2026 年 9 月 19 日
FDA 已受理 UX111（rebisufligene etisparvovec）重新提交的 BLA，用於 MPS IIIA 的加速核准，並指定 PDUFA 行動日期為 2026 年 9 月 19 日。重新提交的資料包含長達八年的追蹤數據，顯示相較於自然病史有持久的臨床改善。
關鍵事實
來源摘錄
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 First Quarter 2026 Financial Results and Corporate Update First quarter total revenue of $136 million, Crysvita® revenue of $93 million and Dojolvi® revenue of $18 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027 GTX-102 for Angelman syndrome: New longer-term data from Phase 1/2 clinical study support durable and improving effects across multiple domains; Phase 3 data expected in the second half of 2026 NOVATO, Calif. – May 05,