原始来源记录
重大申请已受理
Ultragenyx：FDA接受UX111 BLA，设定PDUFA日期为2026年9月19日
FDA接受了UX111（rebisufligene etisparvovec）重新提交的BLA，用于MPS IIIA的加速批准，并指定了2026年9月19日的PDUFA行动日期。重新提交包含长达八年的随访数据，显示出与自然病史相比的持久临床改善。
关键事实
来源摘录
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,