Ultragenyx Pharmaceutical (RARE) said Thursday it submitted a biologics license application to the US Food and Drug Administration for accelerated approval for UX111 to treat Sanfilippo syndrome type A, a lysosomal storage disease that mainly affects the brain.
The company said the submission is supported by available data, including from a study that showed "rapid and sustained decreased levels" of heparan sulfate in cerebral spinal fluid in patients treated with UX111.
Ultragenyx shares were down 1.9% in recent trading.
Price: 42.30, Change: -0.64, Percent Change: -1.49
免責聲明：投資有風險，本文並非投資建議，以上內容不應被視為任何金融產品的購買或出售要約、建議或邀請，作者或其他用戶的任何相關討論、評論或帖子也不應被視為此類內容。本文僅供一般參考，不考慮您的個人投資目標、財務狀況或需求。TTM對信息的準確性和完整性不承擔任何責任或保證，投資者應自行研究並在投資前尋求專業建議。