Posted by Michael Wonder on 10 Dec 2025
FDA grants breakthrough therapy designation to investigational drug adrabetadex for individuals with infantile onset Niemann-Pick disease type C
9 December 2025 - Breakthrough therapy designation is based on FDA’s review of survival analyses comparing adrabetadex-treated patients with external controls and marks a significant regulatory milestone ahead of an NDA submission.
Beren Therapeutics today announced that Mandos received breakthrough therapy designation from the US FDA for adrabetadex, an investigational drug for infantile-onset Niemann-Pick disease type C.
