Posted by Michael Wonder on 23 Feb 2026
Beren Therapeutics announces FDA acceptance of its new drug application for adrabetadex in infantile-onset Niemann-Pick disease type C
23 February 2026 - Beren Therapeutics today announced that the US FDA has accepted for review its new drug application for adrabetadex, an investigational cyclodextrin therapy designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C.
If approved, adrabetadex would represent a first in class, disease-modifying approach to treat infantile-onset Niemann-Pick disease, a rare and rapidly fatal paediatric neurodegenerative disorder.
The FDA assigned adrabetadex a PDUFA target action date of 17 August 2026.
