The U.S. Food and Drug Administration (FDA) granted approval for trofinetide for the treatment of Rett syndrome in adults and children 2 years of age and older. With this approval, trofinetide becomes the first treatment option for this indication. Since treatment options for this condition are limited, experts hope that this option can help fill current gaps, as well as open avenues for other potential specific treatments for this condition.
The approval was based on the results of the phase III LAVENDER trial. You can read more about the efficacy and safety of trofinetide in this trial, as well as its rationale and ongoing trials in our prior articles.
- FDA press release. “FDA approves first treatment for Rett Syndrome.” March 13, 2023. Available at https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-rett-syndrome, accessed March 27, 2023.