Ganaxolone for use in treating tuberous sclerosis complex and seizure disorders
The disclosure to methods for treating tuberous sclerosis complex and epilepsy disorders comprising administering to a subject in need thereof a therapeutically effective amount of ganaxolone or a pharmaceutically acceptable salt thereof.
1. A method for treating a seizure or epilepsy disorder, comprising orally administering to a subject in need thereof ganaxolone in an amount of:
about 150 mg per day for about one week, followed by about 300 mg per day for about one week, followed by about 600 mg per day for about one week, followed by about 1,200 mg per day for about one week, followed by about up to 1,800 mg per day for the remaining treatment period.
2. The method of claim 1 , wherein the subject is concomitantly being administered cannabidiol.
3. The method of claim 1 , wherein about 50 mg are administered three times a day for about one week, followed by about 100 mg administered three times a day for about one week, followed by about 200 mg administered three times a day for about one week, followed by about 400 mg administered three times a day for about one week, followed by 600 mg administered three times a day for the remaining treatment period.
4. The method of claim 1 , wherein ganaxolone is administered as an oral suspension or oral solution.
5. The method of claim 1 , wherein ganaxolone is administered as an oral capsule or tablet.
6. The method of claim 1 , wherein the treatment period is for at least about 5 weeks or longer.
7. The method of claim 1 , wherein administration of ganaxolone does not result in somnolence in the subject.
8. The method of claim 1 , wherein the epilepsy disorder is a focal seizure, a generalized seizure, progressive myoclonic epilepsy, reflex epilepsy, Landau-Kleffner Syndrome, Ohtahara syndrome, Rasmussen's syndrome, infantile spasms (or West syndrome), Lennox-Gastaut syndrome (LGS), Rett syndrome, Dravet syndrome, Doose syndrome, CDKL5 deficiency disorder, tuberous sclerosis complex, intractable childhood epilepsy (ICE), childhood absence epilepsy (CAE), juvenile myoclonic epilepsy (JME), essential tremor, acute repetitive seizures, benign rolandic epilepsy, status epilepticus, refractory status epilepticus, super-refractory status epilepticus, PCDH19 pediatric epilepsy, Angelman syndrome, Doose Syndrome, Aicardi syndrome increased seizure activity, a breakthrough seizures or an infantile spasms.
9. The method of claim 1 , wherein administering ganaxolone reduces the frequency of seizure in the subject relative to baseline.
10. The method of claim 1 , wherein administering ganaxolone results in a reduction in seizure frequency of about 20% or greater relative to baseline seizure frequency.
11. A method for treating a seizure or an epilepsy disorder, comprising orally administering to a subject in need thereof ganaxolone in an amount of:
about 6 mg/kg/day for about one week, followed by about 12 mg/kg/day for about one week, followed by about 24 mg/kg/day for about one week, followed by about 42 mg/kg/day for about one week, followed by about up to 63 mg/kg/day for the remaining treatment period.
12. The method of claim 11 , wherein about 2 mg/kg are administered three times a day for about one week, followed by about 4 mg/kg administered three times a day for about one week, followed by about 8 mg/kg administered three times a day for about one week, followed by about 14 mg/kg administered three times a day for about one week, followed by 21 mg/kg administered three times a day for the remaining treatment period.
13. The method of claim 11 , wherein ganaxolone is administered as an oral suspension or oral solution.
14. The method of claim 11 , wherein ganaxolone is administered as an oral capsule or tablet.
15. The method of claim 11 , wherein the treatment period is for at least about 5 weeks or longer.
16. The method of claim 11 , wherein administration of ganaxolone does not result in somnolence in the subject.
17. The method of claim 11 , wherein the epilepsy disorder is a focal seizure, a generalized seizure, progressive myoclonic epilepsy, reflex epilepsy, Landau-Kleffner Syndrome, Ohtahara syndrome, Rasmussen's syndrome, infantile spasms (or West syndrome), Lennox-Gastaut syndrome (LGS), Rett syndrome, Dravet syndrome, Doose syndrome, CDKL5 deficiency disorder, tuberous sclerosis complex, intractable childhood epilepsy (ICE), childhood absence epilepsy (CAE), juvenile myoclonic epilepsy (JME), essential tremor, acute repetitive seizures, benign rolandic epilepsy, status epilepticus, refractory status epilepticus, super-refractory status epilepticus, PCDH19 pediatric epilepsy, Angelman syndrome, Doose Syndrome, Aicardi syndrome increased seizure activity, a breakthrough seizures or an infantile spasms.
18. The method of claim 11 , wherein administering ganaxolone reduces the frequency of seizure in the subject relative to baseline.
19. The method of claim 11 , wherein administering ganaxolone results in a reduction in seizure frequency of about 20% or greater relative to baseline seizure frequency.
20. The method of claim 1 , wherein the subject weighs more than 28 Kg.
21. The method of claim 3 , wherein the subject weighs more than 28 Kg.
22. The method of claim 11 , wherein the subject weighs 28 Kg or less.
23. The method of claim 12 , wherein the subject weighs 28 Kg or less.
24. The method of claim 11 , wherein the subject is concomitantly being administered cannabidiol.