This week the FDA has stated formally that they see promise in the use of Cyclodextrin in treating Niemann-Pick Type C (NPC) by granting its “Orphan Drug Designation”.

In January of 1983, the Orphan Drug Act (ODA) was passed into law in the United States with consistent pursuit by the National Organization for Rare Disorders. The ideal behind this is to encourage pharmaceutical companies to look into smaller markets with development of drugs for orphan diseases. Niemann-Pick Type C (NPC) certainly falls into this category (around than 500 diagnosed ever worldwide). The benchmark on how to determine an orphan disease is one that affects less than 200,000 people. Continue Reading