for infantile-onset Niemann-Pick disease type C
Originally AUG 17 2026; extended to NOV 17 2026 (FDA classified Beren's response to a March 18, 2026 information request as a Major Amendment to the NDA, extending the review period by three months under standard PDUFA regulations., announced MAY 28 2026).
Infantile-onset Niemann-Pick disease type C is a rare, progressive, and fatal genetic disorder that causes severe neurological decline in early childhood. Adrabetadex is a cyclodextrin-based drug designed to be delivered directly into the spinal fluid, where it is thought to help clear the harmful cholesterol buildup in brain cells. This approach could potentially slow the neurological damage and extend survival for affected children.
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords)
Source: ClinicalTrials.gov