Beren Therapeutics has received FDA approval for review of its application for approval of adrabetadex for the treatment of infantile-onset Niemann-Pick type C disease.[1][2] Adrabetadex is an experimental therapy that improves cholesterol transport in cells and could be the first drug to slow the course of this rare and rapidly progressive childhood neurodegenerative disease.[1][2] The FDA set a decision deadline of August 17, 2026 and assigned the drug priority review status.[1][2] Clinical data show that adrabetadex provides a clinically significant benefit in survival for patients with infantile-onset NPC compared to external controls.[1][2] The drug is generally well tolerated, with the main adverse effects being hearing impairment that can be managed with hearing aids and post-administration fatigue or ataxia.[1] Adrabetadex previously received breakthrough therapy designation in 2025 based on early evidence of significant improvement over existing treatments.[1] The drug is not yet approved by any health regulatory body in the world.[1]