METHODS FOR INCREASING GROWTH IN PEDIATRIC SUBJECTS HAVING CHOLESTATIC LIVER DISEASE
Provided herein are methods for increasing growth in a pediatric subject having a cholestatic liver disease. The method includes administering to the subject an effective amount of an ASBTI.
1 . A method for increasing growth in a pediatric subject having a cholestatic liver disease, the method comprising administering to the subject an effective amount of an ASBTI, wherein the ASBTI is selected from the group consisting of
or a pharmaceutically acceptable salt thereof.
2 . (canceled)
3 . The method of claim 1 , wherein the ASBTI is
or a pharmaceutically acceptable alternative salt thereof.
4 . The method of claim 1 , wherein the ASBTI is volixibat, or a pharmaceutically acceptable salt thereof.
5 .- 7 . (canceled)
8 . The method of claim 1 , wherein the cholestatic liver disease is progressive familial intrahepatic cholestasis (PFIC), biliary atresia, Alagille syndrome (ALGS), or any pediatric cholestatic condition resulting in below normal growth, height, or weight.
9 . The method of claim 1 , wherein the cholestatic liver disease is biliary atresia.
10 . The method of claim 1 , wherein the cholestatic liver disease is PFIC.
11 . The method of claim 10 , wherein the PFIC is selected from PFIC type 1, PFIC type 2, and PFIC type 3.
12 . The method of claim 11 , wherein the PFIC is PFIC type 2.
13 . The method of claim 12 , wherein the subject has an ABCB11 gene with a missense mutation and no truncating mutation.
14 . The method of claim 1 , wherein the cholestatic liver disease is ALGS.
15 . (canceled)
16 . The method of claim 1 , wherein the ASBTI is administered once daily (QD).
17 . The method of claim 16 , wherein the ASBTI is administered in an amount of about 140 μg/kg/day to about 1400 μg/kg/day.
18 . The method of claim 1 , wherein the ASBTI is administered twice daily (BID).
19 . The method of claim 18 , wherein the ASBTI is administered in an amount of about 70 μg/kg to about 700 μg/kg per dose.
20 . The method of claim 1 , wherein an increase in growth is measured as an increase in height or weight Z-score.
21 . The method of claim 20 , wherein the administration results in an increase in height or weight Z-score of at least 0.1 relative to baseline.
22 . The method of claim 21 , wherein the administration results in an increase in height or weight Z-score of at least 0.25 relative to baseline.
23 . The method of claim 1 , wherein the increase in growth is observed within 1 year of first administration of the ASBTI.
24 . The method of claim 1 , wherein an increase in height or weight Z-score is maintained over a period of up to 20 weeks.
25 . The method of claim 24 , wherein the increase in height or weight Z-score is maintained over a period of up to 2 years.
26 .- 37 . (canceled)