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 disease in a human subject in need thereof comprising intrathecally, intravenously, or intracerebroventricularly administering a composition comprising mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises less than 2.5% beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), wherein the composition is suitable for administration to a patient in need thereof, wherein the composition comprises less than 5 IU endotoxin per gram of the beta-cyclodextrin mixture, and wherein the composition comprises no more than 0.5% propylene glycol.
A method of treating Niemann Pick disease in a human subject in need thereof comprising intrathecally, intravenously, or intracerebroventricularly administering a composition comprising mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises less than 2.0% unsubstituted beta-cyclodextrin (“DS-0”);
- and wherein the composition comprises less than 5 IU endotoxin per gram of the beta-cyclodextrin mixture.
A method of treating Niemann Pick disease in a human subject in need thereof comprising administering a composition comprising mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises less than 2.5% beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), wherein the composition is suitable for intrathecal, intravenous, or intracerebroventricular administration to a patient in need thereof, wherein the composition comprises less than 5 IU endotoxin per gram of the beta-cyclodextrin mixture, and wherein the composition comprises no more than 0.2% propylene glycol.
A method of treating Niemann Pick disease in a human subject in need thereof comprising administering a composition comprising mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, wherein:
- the mixture comprises less than 2.0% unsubstituted beta-cyclodextrin (“DS-0”), wherein the composition is suitable for intrathecal, intravenous, or intracerebroventricular administration to a patient in need thereof, wherein the composition comprises no more than 0.5% propylene glycol, and wherein the composition comprises less than 5 IU endotoxin per gram of the beta-cyclodextrin mixture.
Dependencies are inferred from the claim text.