Trofinetide

CHEMBL197084 Phase 4 Zugelassen Small molecule
Half-Life
Bioavailability
Protein Binding
Molecular Weight
315.3 g/mol
LogP
-3.9
Phase
4

Trofinetide is a synthetic analog of glycine-proline-glutamate (GPE), a naturally occurring N-terminal tripeptide fragment of insulin-like growth factor-1, that modulates neuroinflammation and synaptic function in the brain. It is approved for the treatment of Rett syndrome in adults and pediatric patients, a rare neurodevelopmental disorder caused by MECP2 gene mutations, where it reduces core symptoms. Its mechanism involves reducing neuroinflammation and supporting synaptic integrity in affected neural circuits.

Molekularmasse

315,3200 g/mol

LogP

-3,90

TPSA

150,00 Ų

Lipinski-Regel der Fünf

Bestanden

Pharmacokinetics (PK)

Pharmacodynamics (PD)

2D-Struktur

SVG PNG

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SMILES

C[C@@]1(C(=O)N[C@@H](CCC(=O)O)C(=O)O)CCCN1C(=O)CN

InChI

InChI=1S/C13H21N3O6/c1-13(5-2-6-16(13)9(17)7-14)12(22)15-8(11(20)21)3-4-10(18)19/h8H,2-7,14H2,1H3,(H,15,22)(H,18,19)(H,20,21)/t8-,13-/m0/s1

Molecular Formula

C13H21N3O6

HBD / HBA

4 / 7

Rotierbare Bindungen

7

Schwere Atome

22

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

Häufig gestellte Fragen

Trofinetide is a synthetic analog of glycine-proline-glutamate (GPE), a naturally occurring N-terminal tripeptide fragment of insulin-like growth factor-1, that modulates neuroinflammation and synaptic function in the brain. It is approved for the treatment of Rett syndrome in adults and pediatric patients, a rare neurodevelopmental disorder caused by MECP2 gene mutations, where it reduces core symptoms. Its mechanism involves reducing neuroinflammation and supporting synaptic integrity in affected neural circuits.

Yes, Trofinetide is an approved drug. It has reached clinical phase 4. It is classified as a Small molecule.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL197084. Open-access bioactivity database.
  • PubChem — National Center for Biotechnology Information (NCBI). CID 11318905. Chemical information database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-03-28.

Medizinischer Haftungsausschluss

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.