Grandbrothers
Acadia Pharmaceuticals (NASDAQ:ACAD) announced late Friday that the FDA greenlighted trofinetide under the brand name Daybue to treat the rare genetic neurodevelopmental disorder Rett syndrome in those aged two years and older.
Daybue becomes the first and only treatment for Rett syndrome with the approval for which Acadia (ACAD) has received a rare pediatric disease priority review voucher.
Trofinetide is designed to reduce neuroinflammation and support synaptic function, thus addressing the core symptoms of Rett syndrome.
In September, the agency accepted Acadia’s (ACAD) new drug application for trofinetide, granting Mar. 12 as the PDUFA date.
The company has backed the marketing application with data from its pivotal Phase 3 Lavender study, which indicated a statistically significant improvement for trofinetide, over placebo for co-primary endpoints based on two clinical scales related to the disease.
Acadia (ACAD), which has partnered with Australia-based Neuren Pharmaceuticals Limited (OTCPK:NURPF) to develop and commercialize trofinetide in North America, expects the treatment to be available by the end of April 2023.
Seeking Alpha contributor Edmund Ingham downgraded Acadia (ACAD) to Hold from Buy in February, citing a recent upsurge in company shares.