Trofinetide MOA (Mechanism of action):
Trofinetide is a synthetic peptide that has been developed as a potential treatment for Rett syndrome, a rare neurodevelopmental disorder that affects mainly girls.
The mechanism of action of Trofinetide (Trofinetide MOA) is not fully understood, but it is believed to involve several different pathways in the brain.
One of the main targets of Trofinetideis the brain-derived neurotrophic factor (BDNF) pathway.
BDNF is a protein that plays a key role in the development and survival of neurons and is known to be reduced in Rett syndrome.
Trofinetide has been shown to increase the production and release of BDNF, which may help to promote the growth and survival of neurons in the brain.
Trofinetide also acts on the gamma-aminobutyric acid (GABA) system in the brain. GABA is the main inhibitory neurotransmitter in the brain and plays a crucial role in regulating the excitability of neurons.
In Rett syndrome, there is a reduction in the levels of GABA, which can lead to hyperexcitability and seizures. Trofinetide has been shown to increase the levels of GABA, which may help to reduce the hyperexcitability of neurons and improve seizure control.
Another mechanism of action of Trofinetide is its effects on microglia, which are immune cells in the brain.
In Rett syndrome, microglia become activated and produce inflammatory molecules, which can contribute to the neurodegeneration and cognitive impairment seen in the disorder.
Trofinetide has been shown to reduce the activation of microglia and the production of inflammatory molecules, which may help to protect neurons and improve cognitive function.
In summary, Trofinetide appears to have multiple mechanisms of action that may help to improve the symptoms of Rett syndrome by promoting the growth and survival of neurons, increasing GABA levels, and reducing inflammation in the brain.
However, further research is needed to fully understand the molecular pathways involved in trofinetide’s effects and to optimize its use as a treatment for Rett syndrome.
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Pharmacodynamics/ Pharmacokinetics of Trofinetide:
Pharmacodynamics | Description |
| Target receptors | Trofinetide targets brain-derived neurotrophic factor (BDNF), gamma-aminobutyric acid (GABA) system, and microglia in the brain. |
| Trofinetide MOA (Mechanism of action) | Trofinetide increases the production and release of BDNF, which promotes the growth and survival of neurons in the brain. It also increases GABA levels, reducing the hyperexcitability of neurons and improving seizure control. Trofinetide reduces the activation of microglia and the production of inflammatory molecules, which helps to protect neurons and improve cognitive function. |
| Effect | Trofinetide is used to treat Rett syndrome, a rare neurodevelopmental disorder mainly in girls. Trofinetide may help to improve the symptoms of Rett syndrome by promoting the growth and survival of neurons, increasing GABA levels, and reducing inflammation in the brain. |
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Pharmacokinetics | Description |
| Route of administration | Trofinetide is administered via subcutaneous injection. |
| Absorption | Trofinetide is rapidly absorbed after subcutaneous injection, with peak plasma concentrations occurring within 30 minutes to 1 hour. |
| Distribution | Trofinetide has a volume of distribution of 0.13 L/kg, indicating that it is mainly distributed in the plasma and extracellular fluid. |
| Metabolism | Trofinetide is metabolized by peptidases in the plasma and tissues. |
| Elimination | Trofinetide has a half-life of approximately 2-3 hours and is primarily eliminated via the kidneys as small peptides and amino acids. The clearance of trofinetide is not affected by age, gender, or hepatic or renal impairment. |