Theranexus and the Beyond Batten Disease Foundation (BBDF) announce that they have obtained Food and Drug Administration (FDA) approval for the design and primary and secondary endpoints of the pivotal Phase 3 study of Batten-1 in Batten disease, following a meeting with the Division of Rare Diseases and Medical Genetics (DRDMG).

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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