• Comprehensive submission package includes evidence of substantially improved survival among adrabetadex-treated patients with infantile-onset Niemann Pick disease type C (NPC), intended to serve as a single adequate and well-controlled study, and confirmatory evidence of biomarkers and nonclinical data demonstrating effects on the underlying pathology • Priority Review was requested on the basis that the …
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Niemann-Pick Type C News
Adrabetadex (Cyclodextrin) receives FDA Breakthrough Therapy Designation for Individuals with Infantile-Onset NPC
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 New Drug Apllication submission. THOUSAND OAKS, Calif.– December 9, 2025 – Beren Therapeutics P.B.C.®, a Public Benefit Corporation and parent company of Mandos LLC®, today announced that Mandos received Breakthrough Therapy Designation …
Great Data On Adrabetadex/Cyclodextrin Released By Mandos Health
Wednesday, 9/10/25 – In advance of the 2025 American Neurological Association (ANA) meeting, Mandos Health releases two scientific abstracts with promising data for the treatment of NPC patients with Adrabetadex. The lead abstract, designated by ANA as an “Abstract of Distinction,” presents data showing a statistically significant survival benefit in patients with infantile-onset NPC who …