MANDOS LLC

FDA Grants Breakthrough Therapy Designation to Adrabetadex for Infantile-Onset Niemann-Pick Disease Type C

- The FDA has granted Breakthrough Therapy Designation to adrabetadex for infantile-onset Niemann-Pick disease type C, based on survival analyses showing improved outcomes compared to external controls. - Adrabetadex directly addresses the underlying pathology of NPC by re-establishing intracellular cholesterol trafficking, marking a potential therapeutic breakthrough for this devastating neurodegenerative disorder. - Beren Therapeutics plans to submit a New Drug Application in the near future, with the drug expected to be eligible for Priority Review given its orphan drug status. - The designation represents a significant regulatory milestone for a disease with extremely poor prognosis, where early-infantile-onset cases have a mean survival of approximately 5.6 years.