Nakumpleto ng Ultragenyx ang rolling BLA submission para sa DTX401 sa GSDIa

Nakumpleto ng Ultragenyx ang rolling BLA submission para sa DTX401 (pariglasgene brecaparvovec) sa FDA para sa GSDIa. Kasama na ngayon sa submission ang final CMC module matapos ma-file ang non-clinical at clinical modules noong Agosto 2025. Sinusuportahan ang application ng Phase 3 GlucoGene data na nagpapakita ng reduced cornstarch intake, maintained euglycemia, at improved quality of life sa 52 treated patients.

Mahahalagang fact

Na-file: Dis 30, 2025, 09:11 PM
Kaganapan: Naisumite ang aplikasyon
Direksyon: Positibo
Kumpanya: RAREUltragenyx
Asset: pariglasgene brecaparvovec
Pinagmulan: SEC 8-K
Kumpiyansa: May source

Sipi mula sa source

8-K false 0001515673 0001515673 2025-12-30 2025-12-30 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): December 30, 2025 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-K

SEC 8-K