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.
Claims18
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 having an average molar substitution of 0.40 to 0.80, wherein the pharmaceutical composition is administered intrathecally or intracerebroventricularly in a series of escalating doses from about 200 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 having an average molar substitution of 0.58 to 0.68, wherein the pharmaceutical composition is administered intrathecally in a series of escalating doses comprising about 400 mg every two weeks for at least two doses, about 600 mg every two weeks for at least two doses, about 750 mg every two weeks for at least two doses, about 900 mg every two weeks for at least two doses, about 1050 mg every two weeks for at least two doses, and about 1200 mg every two weeks.
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 having an average molar substitution of 0.58 to 0.68, wherein the pharmaceutical composition is administered intrathecally in a series of escalating doses comprising about 200 mg every two weeks for at least two doses, about 300 mg every two weeks for at least two doses, about 400 mg every two weeks for at least two doses, about 500 mg every two weeks for at least two doses, about 600 mg every two weeks for at least two doses, about 700 mg every two weeks for at least two doses, about 800 mg every two weeks for at least two doses, about 900 mg every two weeks for at least two doses, about 100 mg every two weeks for at least two doses, about 1100 mg every two weeks for at least two doses, and about 1200 mg every two weeks.
Dependencies are inferred from the claim text.