Ultragenyx Plans To File For Accelerated Approval Of UX111 For The Treatment Of Sanfilippo Syndrome Type A, After Meeting With FDA
Portfolio Pulse from Benzinga Newsdesk
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) plans to file for accelerated approval of UX111 for the treatment of Sanfilippo Syndrome Type A after a successful meeting with the FDA. The FDA agreed that cerebral spinal fluid heparan sulfate can be used as a surrogate endpoint for the biologics license application.

June 12, 2024 | 8:01 pm
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Ultragenyx Pharmaceutical Inc. (RARE) plans to file for accelerated approval of UX111 for Sanfilippo Syndrome Type A after a successful meeting with the FDA. The FDA agreed that cerebral spinal fluid heparan sulfate can be used as a surrogate endpoint for the biologics license application.
The agreement with the FDA on the use of a surrogate endpoint for UX111's accelerated approval is a significant regulatory milestone. This increases the likelihood of UX111's approval, which could positively impact Ultragenyx's stock price in the short term.
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