原始出處紀錄
決策級FDA 核准
Ultragenyx 獲得 FDA 核准 FAYUVI (UX111) 用於 MPS IIIA
FDA 授予 FAYUVI (rebisufligene etisparvovec-hopf / UX111) 標準完全核准，用於 MPS IIIA (Sanfilippo 症候群 A 型) 兒童患者。這是該適應症首個獲核准的治療，也是 Ultragenyx 第二個基因療法核准。該公司同時獲得優先審查憑證。
關鍵事實
來源摘錄
8-K false 0001515673 0001515673 2026-09-17 2026-09-17 UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): September 17, 2026 Ultragenyx Pharmaceutical Inc. (Exact name of Registrant as Specified in Its Charter) Delaware 001-36276 27-2546083 (State or Other Jurisdiction of Incorporation) (Commission File Number) (IRS Employer Identification No.) 60 Leveroni Court Novato , California 94949 (Address of Principal Executive Offices) (Zip Code) Registrant’s Telephone Number, Including Area Code: 415 483-8800 (Former Name or Former Address, if Changed Since Last Report) Check the appropriate box below if the Form 8-