2010, FDA APPROVAL FOR AN “EMPTY” CYCLODEXTRIN; A MILESTONE IN THE CYCLODEXTRIN RESEARCH AND DEVELOPMENT

摘要

The greatest achievement since the pharmaceutical application of cyclodextrins has started: FDA approval for a cyclodextrin as an Investigational New Drug was received in this year. The so far well known excipient, hydroxypropyl β-cyclodextrin became an active pharmaceutical ingredient (API). The readers of the CDN have been regularly informed on the progress of treatment of six year old identical twin girls dying of a rare brain-destroying cholesterol disease called Niemann Pick Type C (NPC). Known as “childhood Alzheimer’s,” NPC is a deadly progressive neurological condition that causes severe dementia and other debilitating symptoms in children. The FDAs approved use of HPBCD marks the first time in medical history that HPBCD will be delivered directly into the brain of a human being in an attempt to arrest a progressive and fatal neurological condition [1].