发明名称 |
Hydroxypropyl Beta-Cyclodextrin Compositions and Methods |
摘要 |
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. |
申请公布号 |
US2016361345(A1) |
申请公布日期 |
2016.12.15 |
申请号 |
US201615178153 |
申请日期 |
2016.06.09 |
申请人 |
Vtesse, Inc. |
发明人 |
Machielse Bernardus Nicolaas;Darling Allan |
分类号 |
A61K31/724;A61K9/08;A61K9/00 |
主分类号 |
A61K31/724 |
代理机构 |
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代理人 |
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主权项 |
1. A mixture of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, wherein:
the mixture comprises less than 1% unsubstituted beta-cyclodextrin (“DS-0”) and beta-cyclodextrin substituted with one hydroxypropyl group (“DS-1”), collectively; the mixture comprises at least 85% beta-cyclodextrin substituted with three hydroxypropyl groups (“DS-3”), beta-cyclodextrin substituted with four hydroxypropyl groups (“DS-4”), beta-cyclodextrin substituted with five hydroxypropyl groups (“DS-5”), and beta-cyclodextrin substituted with six hydroxypropyl groups (“DS-6”), collectively; the mixture comprises less than 1% beta-cyclodextrin substituted with nine hydroxypropyl groups (“DS-9”) and beta-cyclodextrin substituted with ten hydroxypropyl groups (“DS-10”), collectively, as determined by peak heights of an electrospray MS spectrum. |
地址 |
Gaithersburg MD US |