Rafael Holdings Files New Drug Application To FDA For Approval Of Remibetadex To Treat Neurovisceral Manifestations Of Niemann-Pick Disease Type C

Rafael Holdings, Inc. (NYSE: RFL) today announced that its subsidiary, Cyclo Therapeutics, LLC, has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval of remibetadex (Trappsol® Cyclo™, previously identified as intravenous hydroxypropyl-β-cyclodextrin), for the proposed indication of the treatment of the neurovisceral manifestations of Niemann-Pick Disease Type C (NPC) in adult and pediatric patients from birth. NPC is a rare, fatal and progressive genetic disorder for which there is no known cure. The Company also announced that the United States Adopted Names (USAN) Council has assigned the nonproprietary name "remibetadex" to the compound known until now as intravenous hydroxypropyl-β-cyclodextrin.

Within 60 days of receiving the application, the FDA will determine whether the application is filed and, if filed, whether Priority Review will be granted. If the application is filed, the FDA will assign a Prescription Drug User Fee Act (PDUFA) target action date.