Acadia Pharmaceuticals Inc. (ACAD) announced the publication of clinical data from the open-label DAFFODIL study, which evaluated the long-term safety and efficacy of DAYBUE or trofinetide in young girls ages 2-4 with Rett syndrome.
The study, which included 15 patients, demonstrated that DAYBUE maintained a similar safety profile in younger pediatric patients as seen in older patients in previous Phase 3 studies.
The DAFFODIL study results supported the FDA and Health Canada approvals of DAYBUE for the treatment of Rett syndrome in patients aged 2 years and older.
These findings align with those from the LAVENDER, LILAC-1, and LILAC-2 studies, further reinforcing the drug's safety and effectiveness.
Common side effects in the DAFFODIL study were diarrhea 80 percent and vomiting 53.3 percent, both of which were mild to moderate in severity.
Notably, the study showed improvements in symptoms, such as communication and hand use, based on caregiver feedback. The data also indicated a continued improvement in patient outcomes from Week 4 to Week 78.
Acadia Pharmaceuticals highlighted that this data is critical for refining treatment strategies and understanding the role of trofinetide in managing Rett syndrome, a rare and debilitating condition primarily affecting females.
The findings support the potential for long-term treatment benefits in younger patients with this challenging neurodevelopmental disorder.
Currently, ACAD is trading at $18.29 up by 0.11 percent on the Nasdaq.
For comments and feedback contact: editorial@rttnews.com
Business News
May 08, 2026 15:50 ET Manufacturing and services sector survey results and labor market data from main economies were the highlight on the economics news front this week. Factory orders and jobs report dominated the news flow in the U.S. Similarly, industrial production data from German garnered attention in Europe. In Asia, purchasing managers’ survey results from China and the central bank decision from Australia were in focus.