February 24, 2024

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Daybue: First and only FDA approved drug for Rett syndrome

Daybue: First and only FDA approved drug for Rett syndrome

Daybue: First and only FDA approved drug for Rett syndrome, and the sixth new drug for rare diseases this year, was approved by FDA.

Recently, Acadia Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Daybue (trofinetide) for the treatment of children and adults with Rett syndrome (Rett syndrome) aged two years and older.

Daybue is the first and only drug approved by the FDA for the treatment of Rett syndrome .

This is also the sixth new drug for rare diseases approved by the FDA this year .


Daybue: First and only FDA approved drug for Rett syndrome



Acadia CEO Steve Davis said, “Today marks an important milestone for the Rett syndrome community and for Acadia.

As the first FDA-approved drug for Rett syndrome, Daybue now has the potential to improve the lives of patients and their families.” Make a meaningful difference because they lack options for treating the diverse, debilitating symptoms caused by Rett syndrome.”


Rett syndrome is a complex, rare neurodevelopmental disorder usually caused by mutations in the MECP2 gene, followed by severe developmental regression with loss of acquired communication skills and purposeful use of the hands.

Symptoms of Rett syndrome may also include the development of hand stereotypes, such as hand wringing and clapping, and abnormal gait.

It is reported that Rett syndrome affects 6,000 to 9,000 patients in the United States, and the number of confirmed patients in the United States is about 4,500.


Trofinetide is a synthetic form of a naturally occurring molecule known as the tripeptide glycine-proline-glutamate (GPE).

The mechanism of action of trofinetide in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase dendritic branching and synaptic plasticity signaling.


The FDA’s approval of Daybue is supported by results from the pivotal Phase 3 LAVENDER study, which evaluated the efficacy and safety of trofinetide versus placebo in 187 women with Rett syndrome aged 5 to 20 years.


Compared to placebo, trofinetide treatment demonstrated statistically significant improvements in two co-primary efficacy endpoints, as measured by the Rett Syndrome Behavior Questionnaire (RSBQ) total score (p=0.018) and the Week 12 clinical overall Measured by change from baseline in Impression of Improvement (CGI-I) scale score (p=0.003).

The RSBQ is a caregiver assessment that assesses a spectrum of symptoms of Rett syndrome, including vocalizations, facial expressions, eye gaze, hand movements (or stereotypes), repetitive behaviors, breathing, nocturnal behavior, and mood.

The CGI-I is a method by which physicians worldwide assess whether a patient’s condition is improving or worsening.


In the study, the most common side effects were diarrhea (82%) and vomiting (29%).







(source:internet, reference only)

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