Trofinetide (100mg)

What is Trofinetide (NNZ-2566)?

A synthetic analog of the N-terminal tripeptide of IGF-1 (glycyl-L-prolyl-L-glutamate, GPE) — the first and only FDA-approved drug for Rett syndrome (DAYBUE®).

Listed vial contents: 100mg. These are supplied quantities, not administration doses. Current pricing and availability are shown in the strength selector.

What mechanisms are being studied?

Trofinetide (NNZ-2566) is a synthetic analog of glycyl-L-prolyl-L-glutamate (GPE), the natural N-terminal tripeptide cleavage product of IGF-1 in the central nervous system. It is mechanistically distinct from intact IGF-1: rather than acting through the IGF-1 receptor, it modulates astrocyte / microglial activation, normalizes dendritic spine density, restores synaptic plasticity, and reduces neuroinflammatory cytokine signaling in animal models of neurodevelopmental injury. The molecule is orally bioavailable, crosses the blood–brain barrier, and is stable to enzymatic degradation that rapidly clears native GPE.

What are the research questions?

• Rett syndrome: FDA-approved March 2023 as DAYBUE® (Acadia Pharmaceuticals) for treatment of Rett syndrome in patients ≥2 years — the first approved therapy for the disorder.
• Fragile X syndrome: Phase 2 trials investigated trofinetide for behavioral and cognitive endpoints in Fragile X.
• Neurodevelopmental research: Used in preclinical models of MeCP2-deficient and other neurodevelopmental disorders to study synaptic-plasticity rescue.
• Traumatic brain injury research: Original development indication; preclinical data demonstrated reductions in lesion volume and improved functional recovery.

What does the published work describe?

Neul JL et al. (Nat Med 2023, LAVENDER trial) — Phase 3 demonstrated statistically significant improvement vs placebo on the Rett Syndrome Behaviour Questionnaire and Clinical Global Impression-Improvement at 12 weeks. FDA approval March 2023.

What are the limits of this evidence?

The research described concerns the cited experimental materials and models, not proof that this supplied vial is a treatment. Cell or animal findings do not establish human benefits. Clinical findings, where cited, apply to the tested formulation and population; they do not validate this research product for human use.

Study references

1. Neul JL et al. Nat Med 2023 — LAVENDER Phase 3
2. Glaze DG et al. Neurology 2019 — Phase 2 Rett
3. Bickerdike MJ et al. J Neurol Sci 2009 — NNZ-2566 TBI preclinical

These are the catalog's existing bibliographic references. No new source verification is implied by this presentation.

How should I check lot documentation?

Match the product, strength and lot to an original analysis document. Literature describes experimental findings; it does not verify the contents of this inventory. Generated sample reports illustrate the reporting flow and are not evidence of testing, purity or accreditation.