Ang Ultragenyx Ay Nakatanggap ng Pag-apruba ng FDA para sa FAYUVI (UX111) sa MPS IIIA

Ang FDA ay nagbigay ng karaniwang buong pag-apruba para sa FAYUVI (rebisufligene etisparvovec-hopf / UX111) para sa mga pediatric na pasyente na may MPS IIIA (Sanfilippo syndrome Type A). Ito ang unang naaprubahang paggamot para sa indikasyon at ang pangalawang pag-apruba ng gene therapy para sa Ultragenyx. Ang kumpanya ay nakatanggap din ng isang Priority Review Voucher.

Mahahalagang fact

Na-file: Set 17, 2026, 07:55 PM
Kaganapan: Pag-apruba ng FDA
Direksyon: Positibo
Kumpanya: RAREUltragenyx
Asset: UX111
Pinagmulan: SEC 8-K
Kumpiyansa: May source
Kalendaryo: Desisyon ng FDA (PDUFA) · Set 19, 2026 · Inaprubahan

Sipi mula sa source

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-

SEC 8-K