Life 4 Liam
Adrabetadex
Beren Therapeutics’ cyclodextrin, given into the spinal fluid. Expanded access has been a lifeline for many children with NPC, and an application is under FDA review. It is not Trappsol Cyclo.
How it works
Adrabetadex is a form of hydroxypropyl-beta-cyclodextrin. The idea is that the ring-shaped molecule can bind cholesterol stuck in the lysosome and help carry it out. Doses are given into the spinal fluid on a regular schedule, so the medicine is aimed at the brain and spinal cord.
Beren Therapeutics (Mandos Health) holds the program. Expanded access is how many families receive it today. An NDA is under FDA review. That review is not an approval.
The other cyclodextrin product is Trappsol Cyclo . Do not treat them as the same medicine.
Who it is for
Children whose NPC clinician offers intrathecal adrabetadex through expanded access. The NDA is under FDA review, with a PDUFA target of 17 November 2026. It is not an approved medicine yet.
What it does not do
It does not fix the NPC1 gene or replace the missing protein. It is a different product, dose, and route from intravenous Trappsol Cyclo. It is not a gene therapy. Access still depends on a specialist and on the program’s rules.
What this treatment does
Four short pictures of the idea. Play through, or tap a step. This is a teaching picture, not a medical simulation.
Step 1 of 4 · The problem
Cholesterol is stuck inside the lysosome
NPC1 cannot move it out. The recycling room fills with trapped fat, including in the brain.
Official information
Educational only. Ask an NPC specialist what is right for a specific child or adult. Official sites and FDA pages are the source of prescribing information.