Posted by Michael Wonder on 20 Dec 2025
Beren, through its subsidiary Mandos, submits new drug application to US FDA for adrabetadex in infantile-pnset NPC
19 December 2025 - Beren Therapeutics today announced that its subsidiary Mandos has submitted a new drug application to the US FDA for adrabetadex, an investigational therapy for the treatment of infantile-onset Niemann-Pick disease type C.
The application was based on data supporting a finding of improved survival in an externally controlled survival analysis, biomarker and nonclinical confirmatory evidence, and patient experience narratives.
