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Ultragenyx: Tinanggap ng FDA ang UX111 BLA, itinakda ang PDUFA date sa Sept 19 2026

Tinanggap ng FDA ang muling isinumiteng BLA para sa UX111 (rebisufligene etisparvovec) para sa accelerated approval sa MPS IIIA at itinalaga ang PDUFA action date na September 19, 2026. Kasama sa muling pagsusumite ang hanggang walong taon ng follow-up data na nagpapakita ng matibay na clinical improvement kumpara sa natural history.

Mahahalagang fact

Na-file
May 5, 2026, 08:06 PM
Kaganapan
Tinanggap ang aplikasyon
Direksyon
Positibo
Kumpanya
RAREUltragenyx
Asset
UX111
Pinagmulan
SEC 8-K
Kumpiyansa
May source
Kalendaryo
Desisyon ng FDA (PDUFA) · Set 19, 2026

Sipi mula sa source

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,

SEC 8-K

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