FDA approval for Rett syndrome

CAR-T Digest - August 2020

Mar 10, 2023: DAYBUE (trofinetide, oral) / Rett Syndrome / Acadia Pharmaceuticals: FDA approved DAYBUE for Rett syndrome

The US FDA has authorized DAYBUE (trofinetide) by Acadia Pharmaceuticals for the treatment of Rett syndrome in adults and children ≥2 years.
DAYBUE is expected to be available in the US by the end of April 2023 and is the first and only approved treatment for Rett syndrome.
Rett syndrome is a rare neurodevelopmental disorder that affects 6,000 to 9,000 patients in the US caused by a genetic mutation on the MECP2 gene.
The approval is based on results from the pivotal Phase 3 LAVENDER study evaluating the efficacy and safety of trofinetide versus placebo in 187 female patients.
- Treatment with DAYBUE demonstrated statistically significant improvement compared to placebo on both co-primary efficacy endpoints based on RSBQ & CGI-I score
In 2018, Acadia and Neuren Pharmaceuticals entered into an exclusive license agreement for the development and marketing of trofinetide in North America for the treatment of Rett syndrome and other applications.
With this approval, Acadia was given a voucher for priority review for a rare pediatric disease that can be used for a future application.
ACADIA has also developed and commercialized the first and only medicine approved for the treatment of hallucinations and delusions linked to Parkinson’s disease psychosis and it is currently pursuing clinical development in dementia-related psychosis, the negative symptoms of schizophrenia, major depressive disorder, and Rett syndrome