Trofinetide (Daybue) marks the first drug to be approved for the treatment of Rett syndrome in children aged 2 years or older. The efficacy and satety were evaluated in a ran-domized, double-blind, 12-week study of patients between 5 and 20 years of age.
it Is an analog of insulin-like growth factor 1 and the possible mechanisms of action include:
– Improvement of aberrant neuronal tunction
– Normalization ot synaptic protein synthesis
– Enhancement of antioxidant response
Dosage: 5 – 12g twice dally based on the weight of the patient.
Route of administration: Oral (or via a gas-trostomy tube)
Drug interactions: CYP3A4 sensitive, OATP1B1 and OATP1B3 substrates may lead to serious toxicities
Ref: https://www.accessdata.fda.gov/drugsatfda_docs/label
/2023/217026s000lbl.pdf, https://www.ncbi.nlm.nih.gov
/pmc/articles/PMC6550493/, https://www.rettsyndrome.org
/trofinetideapproved/
