Daybue FDA Approval History
Last updated by Judith Stewart, BPharm on March 16, 2023.
FDA Approved: Yes (First approved March 10, 2023)
Brand name: Daybue
Generic name: trofinetide
Dosage form: Oral Solution
Company: Acadia Pharmaceuticals, Inc.
Treatment for: Rett Syndrome
Daybue (trofinetide) is a synthetic analog of the amino‐terminal tripeptide of IGF-1 for the treatment of Rett syndrome.
- Daybue is approved for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
- Rett syndrome is a rare, debilitating neurological disorder caused by mutations on the X chromosome on a gene called MECP2. Rett syndrome causes problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. Symptoms include loss of fine motor skills, impaired gross motor skills, loss of communication skills (including eye contact), and inability to independently conduct activities of daily living. Symptoms also include seizures, disorganized breathing patterns, scoliosis, and sleep disturbances.
- Daybue is a synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function. IGF-1 is produced by neurons and glial cells in the brain, and is critical for both normal development and for response to injury and disease. Daybue works to treat Rett syndrome by inhibiting the production of inflammatory cytokines, inhibiting the overactivation of microglia and astrocytes, and increasing the amount of available IGF-1 that can bind to IGF-1 receptors.
- Daybue oral solution is administered twice daily, morning and evening, according to patient weight.
- Warnings and precautions associated with Daybue include diarrhea and weight loss.
- Common adverse reactions include diarrhea and vomiting.
Development timeline for Daybue
Further information
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