Ultragenyx: itinakda ang petsa ng PDUFA ng DTX401 sa Q3 2026

BLA rolling submission for DTX401 was completed in December 2025. The FDA has now assigned a PDUFA target action date in the third quarter of 2026, establishing a clear regulatory timeline for potential approval of this AAV gene therapy for GSDIa.

Mahahalagang fact

Na-file: Ene 12, 2026, 12:45 PM
Kaganapan: Naitakda ang petsa ng PDUFA
Direksyon: Positibo
Kumpanya: RAREUltragenyx
Asset: pariglasgene brecaparvovec
Pinagmulan: SEC 8-K
Kumpiyansa: May source
Kalendaryo: Desisyon ng FDA (PDUFA) · Ago 23, 2026 · Inaprubahan

Sipi mula sa source

EX-99.1 2 rare-ex99_1.htm EX-99.1 EX-99.1 Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investments of approximately $735 million as of December 31, 2025 Anticipated 2026 catalysts include two potential approvals and pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif. – January 12, 2026 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2025 revenue results, cash and investments at year end 2025, and p

SEC 8-K