METHOD OF TREATING SYMPTOMS OF PRADER-WILLI SYNDROME
The present disclosure relates generally methods of treating symptoms of Prader-Willi syndrome (PWS), including excessive daytime sleepiness (EDS), hyperphagia, and/or behavioral problems, comprising administering to a subject with PWS a therapeutically effective amount of pitolisant or a pharmaceutically acceptable salt thereof.
1 . A method of treating excessive daytime sleepiness (EDS) in a subject with Prader-Willi syndrome (PWS), comprising administering to a subject with PWS a therapeutically effective amount of pitolisant or a pharmaceutically acceptable salt thereof.
2 . The method of claim 1 , wherein the subject is administered pitolisant monohydrochloride.
3 . The method of claim 1 , wherein the subject is administered WAKIX®.
4 . The method of claim 1 , wherein the therapeutically effective amount comprises a dose of about 1 mg to about 50 mg of pitolisant, wherein mass is based on the freebase form of pitolisant.
5 . The method of claim 1 , wherein the therapeutically effective amount comprises a dose of about 4.45 mg, about 8.9 mg, about 17.8 mg, about 22.25 mg, about 35.6 mg, or about 44.5 mg pitolisant, wherein mass is based on the freebase form of pitolisant.
6 . The method of claim 1 , wherein the method comprises administering an initial dose to the subject that is increased (e.g., doubled) each week for a period of 1-3 weeks after the initial dose is administered, to attain a maintenance dose.
7 . The method of claim 1 , wherein the subject with PWS weighs greater than 40 kg and less than or equal to 80 kg.
8 . The method of claim 7 , wherein the subject is administered an initial dose of about 4.45 mg or about 8.9 mg, optionally increasing to about 8.9 mg or about 17.8 mg at about 1 week after the initial dose is administered, and optionally increasing to a maintenance dose of about 17.8 mg or about 35.6 mg at about 2 weeks after the initial dose is administered.
9 . The method of claim 1 , wherein the subject with PWS weighs greater than 80 kg.
10 . The method of claim 9 , wherein the subject is administered an initial dose of about 4.45 mg or about 8.9 mg, optionally increasing to about 8.9 mg or about 17.8 mg at about 1 week after the initial dose is administered, optionally increasing to about 17.8 mg or about 35.6 mg at about 2 weeks after the initial dose is administered, and optionally increasing to a maintenance dose of about 22.25 mg or about 44.5 mg at about 3 weeks after the initial dose is administered.
11 . The method of claim 1 , wherein the subject with PWS weighs less than or equal to 40 kg.
12 . The method of claim 11 , wherein the subject is administered an initial dose of about 4.45 mg or about 8.9 mg, optionally increasing to a maintenance dose of about 8.9 mg or about 17.8 mg at about 1 week after the initial dose is administered.
13 . The method of claim 1 , wherein the subject with PWS is an adult.
14 . The method of claim 1 , wherein the subject with PWS is an adolescent.
15 . The method of claim 1 , wherein the subject with PWS is a child.
16 . The method of claim 1 , wherein the subject's propensity for sleep is improved, as determined by a validated measurement for sleep propensity.
17 . The method of claim 1 , wherein the subject's Caregiver Global Impression of Severity (CaGI-S) score is reduced.
18 . The method of claim 1 , further comprising reducing hyperphagia in the subject.
19 . The method of claim 1 , further comprising reducing irritability in the subject.
20 . The method of claim 1 , further comprising reducing social withdrawal in the subject.
21 . The method of claim 1 , further comprising reducing hyperactivity/noncompliance in the subject.
22 . The method of claim 1 , further comprising reducing inappropriate speech in the subject.
23 . The method of claim 1 , further comprising reducing stereotypic behavior in the subject.