The Food and Drug Administration has delayed its decision date for Beren Therapeutics’ intrathecal injection intended to treat infantile-onset Niemann-Pick disease type C, according to a company release on May 29. The agency had originally accepted Beren’s data package for priority review in February, setting an initial target action date of August 17. In March, Beren submitted additional data and documentation in response to a previous FDA request.
The FDA considered this new submission a major adjustment to the original drug application, which required an extension of the review period. The new decision date is now set for November 17.
Niemann-Pick disease type C is described as a rare and severe neurodegenerative disorder caused by pathogenic mutations in the NPC1 or NPC2 genes. These mutations impair cholesterol movement inside cells, leading patients to experience progressive neurological decline and early death.
Beren’s candidate drug, adrabetadex, is a proprietary mixture of various isomers of the oligomer 2-hydroxypropyl-β-cyclodextrin. According to the company, this compound can restore cholesterol trafficking within cells. The therapy targets infantile-onset NPC—cases that manifest in children younger than six years old—which typically progress more rapidly and have worse prognoses.
In its February news release, Beren said that adrabetadex reduced the risk of death by 71% compared with external controls and described this effect as highly statistically significant. The FDA granted adrabetadex Breakthrough Therapy designation in December last year.
If approved, adrabetadex would join Zevra Therapeutics’ Miplyffa—which became the first-ever approved treatment for NPC in September 2024—and IntraBio’s Aqneursa on the market. Azafaros is also developing nizubaglustat for NPC; that molecule entered late-stage development last July.
