Acadia, Neuren Headed To Phase III With Trofinetide In Rett Syndrome
Positive Phase II data in the unmet medical need could mean patients will have a therapy specific to their disease, rather than burdensome symptom-by-symptom treatment.
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Acadia’s Daybue Gets First Approval In Rett Syndrome
Daybue is the first drug approved to treat the rare neurodevelopmental disorder Rett syndrome, despite high clinical trial incidence of diarrhea. Product labeling includes clinician guidance for the side effect.
Newron Not Seeing Sarizotan STARS Yet
The Italian firm has some talking to do with the US FDA before it can unblind Phase III data on sarizotan, its Rett syndrome therapy which could be "potentially transformative" for Newron.
Deal Watch, Licensing Focus: Roche, Sanofi Exit Ongoing Anticalin Development Efforts With Pieris
Sarepta licenses gene therapy candidate from Lacerta, also makes equity investment. Neuren licenses North American rights to rare disease drug trofinetide to Acadia.