Beren Therapeutics Receives Breakthrough Therapy Designation for Investigational Drug Adrabetadex for Infantile-Onset Niemann-Pick Disease Type C
Thursday, December 11, 2025
Beren Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to adrabetadex, an investigational treatment for infantile-onset Niemann-Pick disease type C (NPC).
Infantile-onset NPC is a rare and severe neurodegenerative condition in which neurological symptoms appear between birth and six years of age. Earlier onset is associated with faster progression and reduced life expectancy, with average survival of around 5.6 years for early-infantile cases and approximately 13.4 years for late-infantile onset. The condition affects multiple organs, with the most serious complications occurring in the brain.
Adrabetadex (VTS-270) is a mixture of 2-hydroxypropyl-β-cyclodextrin isomers designed to restore intracellular cholesterol transport, directly addressing the underlying cause of NPC. The treatment has generally shown acceptable safety, with common side effects including manageable hearing impairment and occasional fatigue or ataxia following dosing.
The designation is intended to accelerate the development of medicines for serious or life-threatening conditions where early clinical evidence indicates the potential for significant benefit over existing options.
The FDA’s decision was based on an externally controlled survival analysis showing that adrabetadex may improve survival in individuals with infantile-onset NPC, supported by additional biomarker and nonclinical findings. Adrabetadex previously received BTD in 2016 under a different sponsor, but the designation was later withdrawn following results from a Phase 2b/3 trial. Since acquiring the programme in 2021, Beren and Mandos have been working closely with the FDA and the NPC community to advance the therapy and develop new evidence that supports the renewed designation.
The therapy also holds Orphan Drug and Rare Paediatric Disease designations. Beren plans to submit a New Drug Application (NDA) soon and expects the treatment to be considered for Priority Review.
Source: businesswire.com
