• 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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FDA
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 …
FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
FDA Press Release Link: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients | FDA By: Patrizia Cavazzoni, MD, Director, Center for Drug Evaluation and Research and Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research An estimated 10,000+ rare diseases affect more than 30 million people – approximately one …