Ultragenyx Aligns With FDA On Phase 3 Study Endpoints For GTX-102; Phase 3 Study To Initiate By Year-End; Successful End-Of-Phase 2 Meeting Supports Plans For Global Double-Blind Sham-Controlled Pivotal Study; Additional Study Planned For Patients With Other Mutations
Portfolio Pulse from Benzinga Newsdesk
Ultragenyx has reached an agreement with the FDA on the endpoints for its Phase 3 study of GTX-102. The Phase 3 study is set to begin by the end of the year, following a successful end-of-Phase 2 meeting. The study will be a global double-blind sham-controlled pivotal study, with an additional study planned for patients with other mutations.
July 17, 2024 | 8:06 pm
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Ultragenyx has aligned with the FDA on the endpoints for its Phase 3 study of GTX-102, with the study set to begin by year-end. This positive regulatory development supports the company's plans for a global pivotal study and an additional study for other mutations.
The alignment with the FDA on Phase 3 study endpoints is a significant regulatory milestone for Ultragenyx. It indicates a clear path forward for the GTX-102 study, which is likely to boost investor confidence and positively impact the stock price in the short term.
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