Treating Progressive Familial Intrahepatic Cholestasis (PFIC) with IBAT Inhibitors
AssigneeAlbireo AB
InventorsPer-Göran GILLBERG, Jan MATTSSON, Pat HORN, Paresh SONI
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Abstract
Provided herein are methods for treating progressive familial intrahepatic cholestasis (PFIC) with an ileal bile acid transport (IBAT) inhibitor such as odevixibat, or a pharmaceutically acceptable salt thereof. Such methods can include reducing mean pruritus score, mean serum bile acid concentration, increasing height, normalizing weight, improving sleep, and improving liver parameters.
Claims21
-30. (canceled)
A method for treating pruritus associated with Alagille Syndrome (ALGS) in a subject in need thereof, the method comprising orally administering to the subject a therapeutically effective amount of a pharmaceutical formulation comprising odevixibat, or a pharmaceutically acceptable salt thereof,
- wherein following administration of the pharmaceutical formulation, the subject exhibits a reduction in pruritus score, and
- wherein the pharmaceutical formulation comprising odevixibat, or a pharmaceutically acceptable salt thereof, comprises a plurality of particles, wherein each particle is between about 0.1 and about 1.5 mm in size and comprises odevixibat, or a pharmaceutically acceptable salt thereof, in an amount of from about 0.1% w/w to about 5.0% w/w based on the total weight of the particle.
A method for treating pruritus associated with Alagille Syndrome (ALGS) in a subject in need thereof, the method comprising orally administering to the subject a therapeutically effective amount of a pharmaceutical formulation comprising odevixibat, or a pharmaceutically acceptable salt thereof,
- wherein following administration of the pharmaceutical formulation, the subject exhibits a reduction in pruritus score, and
- wherein the pharmaceutical formulation comprising odevixibat, or a pharmaceutically acceptable salt thereof, comprises a plurality of particles, wherein each particle comprises odevixibat, or a pharmaceutically acceptable salt thereof, and each particle of the formulation substantially contains the same amount of odevixibat.
Dependencies are inferred from the claim text.