Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C
Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for adrabetadex, an investigational cyclodextrin therapy designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C (NPC). If approved, adrabetadex would represent a first-in-class, disease-modifying approach to treat infantile-onset NPC, a rare and rapidly fatal pediatric neurodegenerative disorder. The FDA assigned adrabetadex a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026.
