Rafael Holdings files FDA New Drug Application for Remibetadex in Niemann-Pick Disease Type C

Rafael Holdings' subsidiary Cyclo Therapeutics has submitted a New Drug Application to the FDA for remibetadex (Trappsol Cyclo) to treat neurovisceral manifestations of Niemann-Pick Disease Type C in adult and pediatric patients, supported by data from the global Phase 3 TransportNPC study.

Detected & updated continuously · Source: Nebula