Active Ingredient: Trofinetide
Trofinetide is indicated for the treatment of neurobehavioural symptoms of Rett syndrome in adults and paediatric patients aged 5 years and older.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Regimen A, in the case that patient age in years is ≥ 5 and patient weight is ≥ 9 kg and patient weight is < 12 kg :
Oral, 5 grams trofinetide, 2 times daily.
Regimen B, in the case that patient age in years is ≥ 5 and patient weight is ≥ 12 kg and patient weight is < 20 kg :
Oral, 6 grams trofinetide, 2 times daily.
Regimen C, in the case that patient age in years is ≥ 5 and patient weight is ≥ 20 kg and patient weight is < 35 kg :
Oral, 8 grams trofinetide, 2 times daily.
Regimen D, in the case that patient age in years is ≥ 5 and patient weight is ≥ 35 kg and patient weight is < 50 kg :
Oral, 10 grams trofinetide, 2 times daily.
Regimen E, in the case that patient age in years is ≥ 5 and patient weight is ≥ 50 kg :
Oral, 12 grams trofinetide, 2 times daily.
Trofinetide should be taken twice daily, morning and evening, according to patient weight as shown in the following table.
Recommended dosage of trofinetide in patients 5 years of age and older:
| Patient weight | Trofinetide dosage |
| 9 kg to less than 12 kg | 5 g twice daily |
| 12 kg to less than 20 kg | 6 g twice daily |
| 20 kg to less than 35 kg | 8 g twice daily |
| 35 kg to less than 50 kg | 10 g twice daily |
| 50 kg or more | 12 g twice daily |
Continued benefit of treatment should be regularly re-assessed.
The impact of dose reduction on the efficacy of trofinetide is uncertain. Following a dose reduction for the management of adverse reactions, reinstatement of the recommended dose should be considered, based on clinical judgement.
Patients should be advised to stop taking laxatives before starting trofinetide. An interruption, a reduction in dose, or discontinuation of trofinetide should be considered if severe diarrhoea occurs or dehydration is suspected.
If vomiting occurs after trofinetide administration, an additional dose should not be taken. Instead, the next scheduled dose should be taken. An interruption, a reduction in dose, or discontinuation of trofinetide should be considered if vomiting is severe or occurs despite medical management.
An interruption, a reduction in dose, or discontinuation of trofinetide should be considered if significant weight loss occurs.
If a dose of trofinetide is missed, it should be taken as soon as possible. If it is time for the next dose, the missed dose should not be taken. Instead, the next scheduled dose should be taken. Doses should never be doubled to make up for a missed dose.
The efficacy and safety of treatment initiation with trofinetide in patients over 20 years have not been yet established. In patients in whom a clear treatment benefit has been demonstrated, treatment may be continued in patients over 20 years.
Clinical studies of trofinetide did not include patients 65 years of age and older to determine whether or not they respond differently from younger patients. This medicinal product is known to be substantially excreted by the kidney. Because elderly patients are more likely to have decreased renal function, it may be useful to monitor renal function.
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