Active Ingredient: Ganaxolone
Ganaxolone is indicated for the adjunctive treatment of epileptic seizures associated with cyclindependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ganaxolone may be continued in patients 18 years of age and older.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Regimen A: In case that patient weight is ≤ 28 kg, oral, 6 milligrams ganaxolone per kilogram of body weight, 3 times daily to meals, over the duration of 1 week. Afterwards, in case that patient weight is ≤ 28 kg, oral, 11 milligrams ganaxolone per kilogram of body weight, 3 times daily to meals, over the duration of 1 week. Afterwards, in case that patient weight is ≤ 28 kg, oral, 16 milligrams ganaxolone per kilogram of body weight, 3 times daily to meals, over the duration of 1 week. Afterwards, in case that patient weight is ≤ 28 kg, oral, 21 milligrams ganaxolone per kilogram of body weight, 3 times daily to meals.
Regimen B: In case that patient weight is ≥ 28 kg, oral, 150 milligrams ganaxolone, 3 times daily to meals, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 28 kg, oral, 300 milligrams ganaxolone, 3 times daily to meals, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 28 kg, oral, 450 milligrams ganaxolone, 3 times daily to meals, over the duration of 1 week. Afterwards, in case that patient weight is ≥ 28 kg, oral, 600 milligrams ganaxolone, 3 times daily to meals.
Ganaxolone should be titrated gradually to achieve individual clinical response and tolerability. Any patient not tolerating the dosing steps shown in the tables below, can be maintained at the lower dose for additional days before advancing to the next dose. If the next dose is still not tolerated, patients can drop back to the previous lower dose.
It is recommended that total daily dose is administered in 3 equal doses throughout the day. If this is not tolerated by a patient, the dose can be adjusted to manage symptoms (e.g., somnolence), provided that the total daily dose is administered.
In adolescents in whom a clear treatment benefit has been demonstrated, treatment may be continued into adulthood. However, treatment initiation in adults is not recommended as efficacy and safety have not yet been established in this population.
The recommended maximum daily dose is 63 mg/kg/day given in three separate doses (every 8 hours). A minimum dose of 33 mg/kg/day is generally required.
The recommended titration schedule for patients weighing 28 kg or less is shown below:
| Week | Dose (given 3 times a day) | mL/kg per single dose | Total daily dose |
|---|---|---|---|
| Week 1 | 6 mg/kg | 0.12 | 18 mg/kg |
| Week 2 | 11 mg/kg | 0.22 | 33 mg/kg |
| Week 3 | 16 mg/kg | 0.32 | 48 mg/kg |
| Week 4 – ongoing | 21 mg/kg | 0.42 | 63 mg/kg |
The recommended maximum daily dose is 1 800 mg per day given in three separate doses (every 8 hours). A minimum dose of 900 mg/day is generally required.
The recommended titration schedule for patients weighing more than 28 kg is shown below:
| Week | Dose (given 3 times a day) | mL per single dose | Total daily dose |
|---|---|---|---|
| Week 1 | 150 mg | 3 | 450 mg |
| Week 2 | 300 mg | 6 | 900 mg |
| Week 3 | 450 mg | 9 | 1 350 mg |
| Week 4 – ongoing | 600 mg | 12 | 1 800 mg |
If ganaxolone must be discontinued, the dose should be decreased gradually. For patients weighing 28 kg or less, the decrease in total daily dose should be 15 mg/kg every four days. For patients weighing more than 28 kg, the decrease in total daily dose should be 450 mg every four days. Ganaxolone may be stopped immediately and without down-titration in the case of an emergency, however, a down-titration is recommended to minimize the risk of increased seizure frequency and status epilepticus.
Missed doses may be taken up to 4 hours before the next scheduled dose. When the next dose is due in less than 4 hours, it is recommended to skip the dose and to continue with the next scheduled dose.
Ganaxolone must be taken with or shortly after meals and each dose should be administered with similar types of food, if possible. Do not mix with food or drinks prior to administration.
Ganaxolone should only be administered using oral dosing syringes for a more accurate dose administration.
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