Hydroxypropyl beta-cyclodextrin compositions and methods
AssigneeMandos LLC
InventorsBernardus Nicolaas Machielse, Allan Darling
Life of this patent
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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.
Claims30
A method of treating Niemann Pick in a human patient in need thereof comprising administering a composition comprising a mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises unsubstituted beta-cyclodextrin (“DS-0”), beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), and from 5% to 25% beta-cyclodextrin substituted with six hydroxypropyl groups (“DS-6”), and wherein the composition comprises less than 5 International Units (IU) of endotoxins per gram.
A method of treating Niemann Pick in a human patient in need thereof comprising administering a composition comprising a mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises unsubstituted beta-cyclodextrin (“DS-0”), beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), and at least 5% beta-cyclodextrin substituted with six hydroxypropyl groups (“DS-6”), and wherein the composition comprises less than 5 International Units (IU) of endotoxins per gram.
A method of treating Niemann Pick in a human patient in need thereof comprising administering a composition comprising a mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises unsubstituted beta-cyclodextrin (“DS-0”), beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), and no more than 25% beta-cyclodextrin substituted with six hydroxypropyl groups (“DS-6”), and where in the composition comprises less than 5 International Units (IU) of endotoxins per gram.
Dependencies are inferred from the claim text.