JPWO2020123900A5 - - Google Patents

Claims (24)

A pharmaceutical composition for treating dyskinesias in cerebral palsy in a human patient, comprising formula (I):

(wherein each position denoted as D has deuterium enrichment of about 10% or more)
or a pharmaceutically acceptable salt thereof . A pharmaceutical composition for treating dyskinesias in cerebral palsy in a human patient, comprising formula (II):

(wherein each position denoted as D has deuterium enrichment of about 10% or more)
or a pharmaceutically acceptable salt thereof. A pharmaceutical composition for treating dyskinesias in cerebral palsy in human patients (ages 6-18) with DCP with major chorea-type movement disorders, comprising formula (I):

(wherein each position denoted as D has deuterium enrichment of about 10% or more)
or a pharmaceutically acceptable salt thereof. 4. The pharmaceutical composition of any one of claims 1-3, wherein the therapeutically effective amount comprises a daily dose of about 6 mg/day to about 48 mg/day in one or two doses. 4. Any one of claims 1-3, wherein the treatment maintains or reduces abnormal involuntary movements associated with dyskinesias in the patient's cerebral palsy compared to the patient's abnormal involuntary movements at baseline. The pharmaceutical composition according to . 6. The pharmaceutical composition according to claim 5 , wherein the abnormal involuntary movements associated with dyskinesias in cerebral palsy are of the dystonic type. 6. The pharmaceutical composition according to claim 5 , wherein the abnormal involuntary movements associated with dyskinesias in cerebral palsy are of the chorea type. 8. The pharmaceutical composition according to any one of claims 1-7 , wherein the human patient is a pediatric patient. Abnormal involuntary movements associated with dyskinesias in cerebral palsy were assessed by:
a) MD-CRS Part II;
b) MD-CRS Part I;
c) CaGI-I; and
d) CGI-I,
9. The pharmaceutical composition according to any one of claims 5-8 , as measured by at least one of 10. The pharmaceutical composition of claim 9 , wherein abnormal involuntary movements associated with dyskinesias in cerebral palsy are reduced by at least 20% compared to the patient's abnormal involuntary movements at baseline. 10. The pharmaceutical composition of Claim 9 , wherein the patient exhibits a reduction in MD-CRS Part II total score of at least 0.5 points compared to baseline. 10. The pharmaceutical composition of claim 9 , wherein the patient exhibits a reduction in MD-CRS Part I total score of at least 2 points compared to baseline. 13. The pharmaceutical composition of any one of claims 5-12 , wherein the patient has a baseline body weight of at least 12 kg. If the patient weighs between 12 kg and less than 17 kg at baseline and the patient has no CYP2D6 disorder, the daily dose of deutetrabenazine administered is about 24 mg or less; or if the patient has CYP2D6 disorder , about 12 mg or less. If the patient weighs between 17 kg and less than 30 kg at baseline and the patient has no CYP2D6 disorder, the daily dose of deutetrabenazine administered is about 30 mg or less; or if the patient has CYP2D6 disorder , about 18 mg or less. If the patient weighs less than 30 kg to less than 40 kg at baseline and the patient does not have a CYP2D6 disorder, the daily dose of deutetrabenazine administered is about 42 mg or less; or if the patient has a CYP2D6 disorder , about 24 mg or less. If the patient weighs 40 kg or more at baseline and the patient does not have a CYP2D6 disorder, the daily dose of deutetrabenazine administered is about 48 mg or less; or if the patient has a CYP2D6 disorder, about 14. The pharmaceutical composition according to claim 13 , which is 36 mg or less. 18. The pharmaceutical composition of any one of claims 1-17 , wherein each position designated D has a deuterium enrichment of about 50% or greater . 19. The pharmaceutical composition of any one of claims 1-18, wherein each position designated D has a deuterium enrichment of 90% or more. 20. The pharmaceutical composition of any one of claims 1-19, wherein each position designated D has a deuterium enrichment of 95% or more. 21. The pharmaceutical composition of any one of claims 1-20, wherein each position designated D has a deuterium enrichment of 98% or more. 22. The pharmaceutical composition of any one of claims 1-21, wherein each position designated D has a deuterium enrichment of 99% or more. 4. The pharmaceutical composition according to claim 3, wherein the patient has had cerebral palsy symptoms since infancy (under 2 years old). 24. The pharmaceutical composition according to claim 23, wherein the patient has non-progressive cerebral palsy (CP) symptoms since infancy.