AUG 23 2026
Beren Therapeutics — Adrabetadex
for infantile-onset Niemann-Pick disease type C
Accepted FEB 23 2026.
About
Infantile-onset Niemann-Pick disease type C is a rare genetic disorder that disrupts cholesterol transport within cells, leading to severe neurological symptoms and early mortality. Adrabetadex is a cyclodextrin-based drug that binds to cholesterol and helps remove its excess buildup in cells. By restoring cholesterol balance, Adrabetadex may slow disease progression and improve neurological function in affected children.
Pivotal trial
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords)
Source: ClinicalTrials.gov
Also in development
Drug Watch — recent news on Adrabetadex
- FDA extends review timeline of Beren’s adrabetadex NDA - The Pharma Letter
- Beren Therapeutics Announces FDA Extension of Review Period for New Drug Application (NDA) for Adrabetadex for the Treatment of Infantile-Onset Niemann-Pick Disease, Type C - Business Wire
- FDA extends adrabetadex NDA review for infantile-onset Niemann-Pick disease type C - Contemporary Pediatrics
- Beren Therapeutics Presents Data Supporting the Efficacy of Adrabetadex in Infantile-Onset NPC at 2026 ACMG Clinical Genetics Meeting - BioSpace
- Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C - Business Wire