Posted by Beren Therapeutics Announced 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.
Key findings were recently presented at WORLDSymposium™ 2026, the largest annual scientific meeting in the field of lysosomal storage disease, including survival data in infantile-onset NPC: a 71% reduction in the risk of mortality in adrabetadex-treated patients compared with matched external controls (HR 0.289; 95% CI, 0.141–0.593; P < 0.0001).
About Adrabetadex
Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation as a treatment for Niemann-Pick disease type C (NPC). The data suggest that by re-establishing intracellular cholesterol trafficking, adrabetadex is designed to directly address the underlying pathology of NPC. Data from clinical trials and expanded access programs suggest that adrabetadex is generally well tolerated. The main adverse events associated with adrabetadex include hearing impairment that can be managed with hearing aids when necessary, and post-dose fatigue and/or ataxia. Adrabetadex has not been approved by the FDA or any other health authority at this time.