Recognizing urgency. moving with rigor. Building a better future.
We partner with patients and families to deeply understand what’s most important and build a path forward.
Our journey with the NPC community so far
The NPC community has been on a long journey to bring adrabetadex to families affected by the disease. We are proud to have been on this journey alongside them since 2021 and to continue to partner with them for decades to come.
2026
- New Drug Application for adrabetadex in infantile-onset Niemann Pick Disease Type C is accepted by the FDA
2025
- Beren submits a New Drug Application for adrabetadex to the FDA through its subsidiary Mandos LLC
- Breakthrough Therapy designation is regranted by the FDA for adrabetadex
- Data analysis suggests a significant survival benefit, which supports a New Drug Application
2022
- Beren held discussions with the FDA to develop a rigorous methodology to support survival analysis in infantile-onset NPC. This drew on over 11 years of clinical data and incorporated the largest, detailed natural history studies and patient registry data of people living with NPC.
2021
- Beren acquires adrabetadex through its subsidiary Mandos LLC
The early history of adrabetadex
2021
- Survival hypothesis proposed by the principal investigator of the expanded access program, Dr. Liz Berry-Kravis
2020
- Mallinckrodt, the previous sponsor of the adrabetadex program enters bankruptcy and terminates the program
2019
- Phase 2b/3 results show no significant difference in co-primary endpoint
2017
- Phase 1/2a results published in Lancet
2013
- NPC Program started clinical studies
- Initiation of RUSH Expanded Access Program for intrathecal cyclodextrin.
2009
- Cyclodextrin approved under compassionate use by the FDA to treat Addison and Cassidy Hempel
- Research published showing effect of cyclodextrins in mouse models of NPC
2007
- NPC families, healthcare providers, and researchers join together to accelerate research on treatments for NPC
