trofinetide (Pending FDA Approval)

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Dosing & Uses

AdultPediatric

Rett Syndrome

Pending FDA approval for treatment of Rett syndrome

Rett Syndrome

Pending FDA approval for treatment of Rett syndrome in adults and children aged ≥2 years

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Pharmacology

Mechanism of Action

Synthetic analog of the amino‐terminal tripeptide of insulin-like growth factor (IGF-1) designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function

IGF-1 is produced by both of the major types of brain cells – neurons and glia; IGF-1 in the brain is critical for both normal development and for response to injury and disease

Trofinetide has been shown to inhibit the production of inflammatory cytokines, inhibit overactivation of microglia and astrocytes, and increase amount of available IGF-1 that can bind to IGF-1 receptors

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Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.