FDA Grants Breakthrough Therapy Designation to Investigational Drug Adrabetadex for Infantile-Onset Niemann-Pick Disease Type C
Wednesday, December 10, 2025
Beren Therapeutics, through its subsidiary Mandos LLC, has received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational treatment for infantile-onset Niemann-Pick disease type C (NPC).
The FDA grants BTD to support the faster development of medicines for serious or life-threatening conditions when early clinical evidence suggests a clear improvement over available therapies. The decision for adrabetadex was based on an externally controlled survival analysis indicating improved survival in individuals with infantile-onset NPC, supported by biomarker findings and nonclinical data.
Adrabetadex previously received BTD in 2016 under a different sponsor, but the designation was withdrawn following results from a Phase 2b/3 trial. Beren, which acquired the programme in 2021, has since worked with the FDA and the NPC community to further develop the therapy and provide additional data to support the renewed designation.
Clinicians noted that infantile-onset NPC is a severe and rapidly progressing condition, and the survival improvements observed so far represent a potential advancement in treatment options.
Adrabetadex also holds Orphan Drug and Rare Paediatric Disease designations. Beren plans to submit a New Drug Application (NDA) soon and anticipates that the therapy may qualify for Priority Review.
Source: businesswire.com
