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trofinetide
N07XX24
Adult Dosing
- Not evaluated or indicated in adult populations; Rett syndrome primarily affects pediatric patients.
Pediatric Dosing
- Rett syndrome (patients 2 years of age and older): Weight-based dosing twice daily, administered orally or via gastrostomy tube.
- 10 kg to < 12 kg: 5 g (25 mL) twice daily
- 12 kg to < 20 kg: 6 g (30 mL) twice daily
- 20 kg to < 35 kg: 8 g (40 mL) twice daily
- 35 kg to < 50 kg: 10 g (50 mL) twice daily
- 50 kg and above: 12 g (60 mL) twice daily
Indications
- Treatment of Rett syndrome in adult and pediatric patients 2 years of age and older
Mechanism of Action
- Synthetic analogue of glycine-proline-glutamate (GPE), a naturally occurring tripeptide derived from insulin-like growth factor 1 (IGF-1).
- Acts to reduce neuroinflammation and support synaptic function in the central nervous system, addressing underlying pathophysiology of Rett syndrome.
Contraindications
- Known hypersensitivity to trofinetide or any of its excipients
Adverse Reactions
- Diarrhea (very common, often requires antidiarrheal management and hydration)
- Vomiting
- Weight decrease
- Decreased appetite
- Pyrexia
- Fatigue
Drug Interactions
- Substrate of OATP1B1 and OATP1B3 transporters; avoid coadministration with strong OATP1B1/1B3 inhibitors.
- Potential to inhibit or induce certain CYP450 enzymes or transporters requires clinical monitoring when coadministered with narrow therapeutic index drugs.
Curated Content: Needs Vetting Before Put to Clinical Use