FDA Approves First Treatment for Rett Syndrome
TUESDAY, March 14, 2023 (HealthDay News) – The U.S. Food and Drug Administration has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older.
Approval was based on results from the phase 3 LAVENDER study in which the efficacy and safety of Daybue was assessed versus placebo in 187 female patients with Rett syndrome (ages 5 to 20 years). Compared with placebo, Daybue was associated with statistically significant improvement for change from baseline in the Rett Syndrome Behaviour Questionnaire total score and the Clinical Global Impression-Improvement scale score at week 12.
The most common side effects were diarrhea (82 percent) and vomiting (29 percent). Daybue is expected to be available in the United States by the end of April 2023.
"As the first FDA-approved drug for the treatment of Rett syndrome, Daybue now offers the potential to make meaningful differences in the lives of patients and their families who have lacked options to treat the diverse and debilitating array of symptoms caused by Rett syndrome," Steve Davis, CEO of Acadia Pharmaceuticals, said in a statement.
Approval of Daybue was granted to Acadia Pharmaceuticals.
Related Posts
Lenvatinib + Pembrolizumab Treats Advanced Endometrial Cancer
MONDAY, Jan. 31, 2022 (HealthDay News) -- For patients with advanced endometrial...
Una terapia ‘pulsada’ para los nervios podría ayudar a aliviar la ciática
MIÉRCOLES, 29 de marzo de 2023 (HealthDay News) -- Las personas que sufren de...
Retinal Layer Thickness Linked to Cognitive Decline in Older Adults
FRIDAY, May 27, 2022 (HealthDay News) -- Macular retinal nerve fiber layer...