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 severe genetic disorder that causes progressive neurological damage, including loss of motor control and cognitive decline. Adrabetadex is a drug designed to reduce the harmful buildup of cholesterol and other lipids inside cells. By clearing these cellular blockages, the treatment could potentially slow the progression of the neurological symptoms that define this condition.
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
- Beren Therapeutics Secures $300 Million Financing To Support Potential Adrabetadex Launch - Pulse 2.0
- Beren Therapeutics Secures $300M Financing to Support the Potential Commercial Launch of Adrabetadex and Long-Term Care Initiatives for Infantile-Onset Niemann-Pick Disease, Type C - finance.yahoo.com
- FDA Extends Adrabetadex Review for Infantile-Onset Niemann-Pick Type C - Neurology Advisor
- 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