Hydroxypropyl beta-cyclodextrin compositions and methods
AssigneeVtesse Inc
InventorsBernardus Nicolaas Machielse, Allan Darling
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Abstract
This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.
Claims14
A method for treating Niemann-Pick disease Type C in a human, the method comprising administering to the human a pharmaceutical composition comprising a mixture of hydroxypropyl beta-cyclodextrin molecules, wherein the mixture of hydroxypropyl beta-cyclodextrin molecules has an average molar substitution of 0.40 to 0.80, and the pharmaceutical composition is administered intrathecally or intracerebroventricularly in a dosage of about 600 mg to about 1500 mg.
A method for treating Niemann-Pick disease Type C in a human, the method comprising administering to the human a pharmaceutical composition comprising a mixture of hydroxypropyl beta-cyclodextrin molecules, wherein the mixture of hydroxypropyl beta-cyclodextrin molecules has an average molar substitution of 0.58 to 0.68, and the pharmaceutical composition is administered intrathecally or intracerebroventricularly in a dosage of 900 mg every two weeks.
Dependencies are inferred from the claim text.