Adrabetadex
An investigational treatment for Niemann-Pick Disease Type C.
The same compound appears under different names depending on the context. Here is how to identify Adrabetadex wherever you encounter it, plus the key facts at a glance.
- Generic name
- Adrabetadex
- Development code
- VTS-270
- Drug class
- Cyclodextrin, given into the spinal fluid
- Manufacturer
- Beren Therapeutics
- How it's taken
- In the Phase 2b/3 trial, adrabetadex was given by lumbar puncture into the spinal fluid every 2 weeks at doses of 900 to 1800 mg, according to the study's ClinicalTrials.
Beren Therapeutics' investigational cyclodextrin for infantile-onset Niemann-Pick disease type C, meaning neurological symptoms that begin before age 6, given into the spinal fluid. The FDA is due to decide on it by November 17, 2026, after extending its review from August 17.
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Where Adrabetadex fits
The 2 FDA-approved NPC drugs, Miplyffa with miglustat from age 2 and Aqneursa from 15 kg body weight, leave most children under 2 without an approved option. If approved for infantile-onset NPC, adrabetadex would be the first NPC drug given into the spinal fluid.
How Adrabetadex works
In Niemann-Pick type C, a faulty NPC1 or NPC2 protein leaves cholesterol stuck inside the cell's recycling compartments, the lysosomes, and nerve cells are damaged over time. Adrabetadex is a mixture of cyclodextrins, ring-shaped sugar molecules that can carry cholesterol out of those compartments, according to Beren. Because NPC damages the brain, the drug is given into the spinal fluid rather than a vein.
Mechanism: A mixture of 2-hydroxypropyl-beta-cyclodextrins that Beren says draws cholesterol out of the lysosomes where it is trapped in NPC cells, restoring cholesterol trafficking
Side effects and safety
Beren lists hearing impairment, which it says can be managed with hearing aids, and tiredness or unsteadiness after a dose as the main adverse events. The NIH reported in 2017 that patients in its early study had additional hearing loss that hearing aids compensated for.
Repeated lumbar punctures add the burden and risks of a procedure every 2 weeks, which for a small child can involve sedation. There is no FDA label yet, so the full list of warnings is not known.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Adrabetadex
In the Phase 2b/3 trial, adrabetadex was given by lumbar puncture into the spinal fluid every 2 weeks at doses of 900 to 1800 mg, according to the study's ClinicalTrials.gov record. We found no dose or schedule for infants in Beren's public announcements; an FDA label would set both.
Clinical trial results
Beren reported in October 2025, and repeated in March 2026, that 72 people with infantile-onset NPC treated with adrabetadex had a 71% lower risk of death than 119 matched untreated people from the NIH natural history study and other databases, with 5-year survival of 84% against 42%. In 79 treated patients, neurological decline was 43% slower in the year after starting than before, Beren reported.
The Phase 2b/3 trial (NCT02534844) randomly assigned 56 people aged 4 to 21 to adrabetadex or a sham procedure. After 52 weeks neither group had changed on the NPC severity score, a change of 0.0 on adrabetadex against minus 0.1 on sham, according to results posted on ClinicalTrials.gov.
Main registered trial: NCT02534844 on ClinicalTrials.gov. Check it for the current status, sites and contacts before asking about enrollment.
Development history
An NIH-led early study, published in The Lancet in 2017, gave the cyclodextrin into the spinal fluid of people with NPC1. The drug then passed from Vtesse to Sucampo and Mallinckrodt, which stopped developing it in January 2021, and Mandos bought it in July 2021. Mandos, now a subsidiary of Beren Therapeutics, submitted a new drug application for infantile-onset NPC in December 2025. The FDA granted Breakthrough Therapy designation in December 2025, having first granted it in 2016 and withdrawn it after the Phase 2b/3 trial, according to Beren, and accepted the application with priority review in February 2026, then moved the goal date from August 17 to November 17, 2026 after classifying a March 2026 response as a major amendment.
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Common questions about Adrabetadex
▸What is Adrabetadex?
Beren Therapeutics' investigational cyclodextrin for infantile-onset Niemann-Pick disease type C, meaning neurological symptoms that begin before age 6, given into the spinal fluid. The FDA is due to decide on it by November 17, 2026, after extending its review from August 17.
▸How does Adrabetadex work?
In Niemann-Pick type C, a faulty NPC1 or NPC2 protein leaves cholesterol stuck inside the cell's recycling compartments, the lysosomes, and nerve cells are damaged over time. Adrabetadex is a mixture of cyclodextrins, ring-shaped sugar molecules that can carry cholesterol out of those compartments, according to Beren. Because NPC damages the brain, the drug is given into the spinal fluid rather than a vein.
▸What are the side effects of Adrabetadex?
Beren lists hearing impairment, which it says can be managed with hearing aids, and tiredness or unsteadiness after a dose as the main adverse events. The NIH reported in 2017 that patients in its early study had additional hearing loss that hearing aids compensated for.
Repeated lumbar punctures add the burden and risks of a procedure every 2 weeks, which for a small child can involve sedation. There is no FDA label yet, so the full list of warnings is not known.
▸How is Adrabetadex taken?
In the Phase 2b/3 trial, adrabetadex was given by lumbar puncture into the spinal fluid every 2 weeks at doses of 900 to 1800 mg, according to the study's ClinicalTrials.gov record. We found no dose or schedule for infants in Beren's public announcements; an FDA label would set both.
▸Is Adrabetadex FDA approved?
Not yet. Adrabetadex is under FDA review for Niemann-Pick Disease Type C; Beren Therapeutics has filed an application and a decision is due November 17, 2026. The FDA calendar page tracks the outcome.
▸When will the FDA decide on adrabetadex?
The FDA's goal date is November 17, 2026, moved from August 17 after the FDA classified a March 2026 response from Beren as a major amendment. The adrabetadex FDA decision page will show the outcome the day it is public.
▸Is adrabetadex FDA approved?
No. Adrabetadex is investigational. Beren Therapeutics has asked the FDA to approve it for infantile-onset Niemann-Pick type C, and a decision is due by November 17, 2026. Beren says its expanded access program continues during the review.
▸Can babies with Niemann-Pick type C get an approved drug?
Most cannot under current labels. Miplyffa is approved from age 2 and Aqneursa from 15 kg body weight, and every NPC treatment trial recruiting on ClinicalTrials.gov starts at age 4, as of October 14, 2026.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Beren Therapeutics · 2026-03-12. Beren Therapeutics presents data supporting the efficacy of adrabetadex in infantile-onset NPC at 2026 ACMG meeting. https://www.berentx.com/news/beren-therapeutics-presents-data-supporting-the-efficacy-of-adrabetadex-in-infantile-onset-npc-at-2026-acmg-clinical-genetics-meeting
- Beren Therapeutics · 2026-05-28. Beren Therapeutics announces FDA extension of review period for adrabetadex NDA. https://www.berentx.com/news/beren-therapeutics-announces-fda-extension
- Beren Therapeutics · 2026-02-23. FDA acceptance of NDA for adrabetadex in infantile-onset NPC. https://www.berentx.com/news/adrabetadex-nda-accepted-priority-review-npc
- ClinicalTrials.gov. VTS-270 to treat Niemann-Pick type C1 disease, results. https://clinicaltrials.gov/study/NCT02534844
- NICHD · 2017-08-09. NIH-led study results show progress against Niemann-Pick disease type C1. https://www.nichd.nih.gov/news/releases/Pages/nieman_pick_080917.aspx
- Business Wire · 2021-07-01. Mandos LLC completes acquisition of adrabetadex from Mallinckrodt Pharmaceuticals. https://www.businesswire.com/news/home/20210701005935/en/Mandos-LLC-Completes-Acquisition-of-Adrabetadex-from-Mallinckrodt-Pharmaceuticals