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 …
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FDA
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 …
NPC Parents Help Get First Drug Recommendation!
A Food and Drug Administration (FDA) advisory panel recommended Zavesca to treat NPC. Phil Marella of DART, Cindy Parseghian of the Parseghian Foundation and Barb Vorpahl of the NNPDF spoke during the public portion of the hearing. The panel of outside medical experts voted 10-to-3 in favor of a question that asked “in light of …