Beren Therapeutics has received the US Food and Drug Administration (FDA) acceptance to review its New Drug Application (NDA) for adrabetadex, an investigational therapy targeting intracellular cholesterol trafficking for infantile-onset Niemann-Pick disease type C (NPC). Adrabetadex is a mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation for NPC.
This marks a key development in the potential approval of adrabetadex, which could become a disease-modifying treatment for this rare paediatric neurodegenerative disorder.
Clinical Data and Regulatory Timeline
PDUFA Date: The FDA set 17 August 2026 as the target action date for the adrabetadex application under the Prescription Drug User Fee Act (PDUFA).
The NDA submission includes data indicating a clinically meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex. This evidence is drawn from an externally controlled analysis designed to serve as a single adequate and well-controlled study. Supporting data show relevant biomarker, nonclinical findings, and slowed disease progression.
In 2025, adrabetadex obtained breakthrough therapy designation from the FDA due to early evidence suggesting substantial improvement over existing treatments. As previously noted, adrabetadex is a combination of 2-hydroxypropyl-β-cyclodextrin isomers under investigation for NPC.
Safety Profile and Tolerability
Data from clinical trials and expanded access programmes suggest it is generally well tolerated, with main adverse events including hearing impairment, manageable with hearing aids, and post-dose fatigue or ataxia. Adrabetadex has not been approved by any health authority to date.
