熱門資訊> 正文

Ultragenyx启动基因疗法的FDA营销申请程序

2025-08-18 21:02

Ultragenyx Pharmaceutical (NASDAQ:RARE) said that it has started the rolling submission of a marketing application to the U.S. FDA for its DTX401 AAV gene therapy, aimed at treating glycogen storage disease Type Ia.
The company has already provided the FDA with the non-clinical and clinical sections of the application.
They plan to finalize the completion of the application by the end of 2025, which will include details on chemistry, manufacturing, and controls.

More on Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. (RARE) Q2 2025 Earnings Call Transcript
Ultragenyx: July Selloff Creates Buying Opportunity Ahead Of UX143 Catalyst
Ultragenyx: The High Stakes Of Binary Events And A Short Cash Runway
Ultragenyx outlines $640M–$670M 2025 revenue target as clinical milestones advance
Ultragenyx Pharmaceutical GAAP EPS of -$1.17 beats by $0.13, revenue of $166.5M beats by $5.13M

推薦文章

風險及免責提示：以上內容僅代表作者的個人立場和觀點，不代表華盛的任何立場，華盛亦無法證實上述內容的真實性、準確性和原創性。投資者在做出任何投資決定前，應結合自身情況，考慮投資產品的風險。必要時，請諮詢專業投資顧問的意見。華盛不提供任何投資建議，對此亦不做任何承諾和保證。