Theranexus: FDA approval for Batten disease
The primary endpoint of phase 3 will be visual acuity, with secondary endpoints including assessment of cognitive and motor function. The FDA has confirmed that this Phase 3 study alone will allow registration of Batten-1 in CLN3 Batten disease.
' We are very pleased with the constructive discussions we had with the FDA on the definition of the endpoints for Batten-1 and the design of our pivotal Phase 3 study. Its recommendations are invaluable to us, and will enable us to prepare the launch of this study in the best possible way", said Marie Sébille, Theranexus' Medical Director.
' Our pivotal Phase 3 study will not only enable the registration of Batten-1, but will also offer a rich newsflow throughout this Phase 3, thanks to a parallel cohort of open-label patients. More broadly, we are convinced that we can provide a therapeutic solution for patients suffering from Batten disease' announced Mathieu Charvériat, CEO of Theranexus.
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