Trofinetide

Synthetic Peptide AnalogRx: PrescriptionCompound: Approved

Also known as: Daybue, Glypromate, GPE analog, GPE analogue, NNZ-2566

Educational Only — Not medical advice. Consult a qualified clinician before using any peptide.

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Summary

Trofinetide is an oral synthetic peptide analog of glycine‑proline‑glutamate approved for the treatment of Rett syndrome in patients aged ≥5 years, improving neurobehavioral function.

Mechanism of Action

Modulates neuroinflammation and synaptic function; enhances microtubule stability and neuronal connectivity

Routes of Administration

Oral

Goals & Uses

  • Fragile X syndromeNeurological DisorderLow
  • Improvement of synaptic functionNeuroscienceModerate
  • Rett syndromeNeurodevelopmental DisorderHigh
  • Treatment of Rett syndromeNeurodevelopmental DisorderHigh
  • Traumatic brain injuryNeuroprotectionLow
  • Reduction of neuroinflammationNeurologyModerate

Contraindications

  • Hypersensitivity to trofinetide or excipientsAllergic/immunologicHigh
  • Hypersensitivity to trofinetideAllergyHigh
  • Severe renal impairmentOrganModerateKidney function concerns

Adverse Effects

  • NasopharyngitisInfectiousCommon
  • HeadacheNeurologicCommonPain in the head or upper neck
  • Weight loss / decreased appetiteMetabolicCommon
  • NauseaGastrointestinalCommonFeeling of sickness or urge to vomit
  • FatigueGeneralUncommonLow energy or tiredness
  • VomitingGastrointestinalCommonForceful expulsion of stomach contents
  • DiarrheaGastrointestinalCommonLoose or frequent stools
  • DehydrationFluid BalanceUncommonLoss of body water or inadequate hydration
  • Abdominal painGastrointestinalCommonPain or discomfort in the abdomen

Drug Interactions

  • Oral contraceptivesModerateDelayed gastric emptying may affect absorption timing
  • none identifiedLow
  • CYP3A4 substrates (sensitive)Moderate

Population Constraints

  • PregnancyReproductive SafetyRelative
  • Age ≥5 yearsAgeAbsolute
  • Children under 2 years of agePediatricAbsolute
  • BreastfeedingReproductiveRelative
  • Severe renal impairment (eGFR <30 mL/min)RenalRelative

Regulatory Status

  • European UnionInvestigationalSubmitted for marketing authorization; pending
  • United StatesApprovedApproved: Rett syndromeApproved as Daybue for patients ≥5 years
  • United KingdomUnknown

FDA approved in August 2023 (brand name Daybue) as the first therapy for Rett syndrome; under regulatory review in the EU.

Evidence & Sources

No sources recorded yet.

Frequently Asked Questions

What is Trofinetide?

Trofinetide is an oral synthetic peptide analog of glycine‑proline‑glutamate approved for the treatment of Rett syndrome in patients aged ≥5 years, improving neurobehavioral function.

What is Trofinetide used for?

Trofinetide is educationally associated with: Fragile X syndrome, Improvement of synaptic function, Rett syndrome, Treatment of Rett syndrome, Traumatic brain injury, Reduction of neuroinflammation. Educational only — not medical advice.

How is Trofinetide administered?

Recorded routes of administration: Oral.

What are the potential side effects of Trofinetide?

Reported adverse effects include: Nasopharyngitis, Headache, Weight loss / decreased appetite, Nausea, Fatigue, Vomiting, Diarrhea, Dehydration, Abdominal pain. This list is not exhaustive — consult a qualified clinician.

Who should avoid Trofinetide?

Recorded contraindications: Hypersensitivity to trofinetide or excipients, Hypersensitivity to trofinetide, Severe renal impairment. Consult a qualified clinician before use.