FDA Grants Clearance for Experimental Intrathecal Administration of Cyclodextrin
The FDA has granted clearance of an Investigational New Drug (IND) application for administration of Hydroxypropyl Beta Cyclodextrin (cyclodextrin) into the central nervous systems of two patients with Niemann-Pick Disease Type C.
The NNPDF would like to note the following regarding cyclodextrin:
1. Cyclodextrin has shown promise as a potential therapeutic in animal trials. However, issues of toxicity have also arisen.
2. The NNPDF is aware that cyclodextrin is being used on a single-patient Investigational New Drug (IND) basis in the U.S. and in Brazil.
3. More research is needed to understand issues related to drug delivery, efficacy and safety.
4. The NNPDF will keep the NPC community apprised of developments regarding cyclodextrin.