Beren Therapeutics has received the US Food and Drug Administration (FDA) acceptance to review its New Drug Application (NDA) for adrabetadex, an investigational therapy targeting intracellular cholesterol trafficking for infantile-onset Niemann-Pick disease type C (NPC).

This marks a key development in the potential approval of adrabetadex, which could become a disease-modifying treatment for this rare paediatric neurodegenerative disorder.

The FDA set 17 August 2026 as the target action date for the adrabetadex application under the Prescription Drug User Fee Act (PDUFA).

The NDA submission includes data indicating a clinically meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex.

This evidence is drawn from an externally controlled analysis designed to serve as a single adequate and well-controlled study. Supporting data show relevant biomarker, nonclinical findings and slowed disease progression.

In 2025, adrabetadex obtained breakthrough therapy designation from the FDA due to early evidence suggesting substantial improvement over existing treatments.

Adrabetadex is a combination of 2-hydroxypropyl-β-cyclodextrin isomers under investigation for NPC.

Data from clinical trials and expanded access programmes suggest it is generally well tolerated, with main adverse events including hearing impairment, manageable with hearing aids, and post-dose fatigue or ataxia. Adrabetadex has not been approved by any health authority.

Beren Therapeutics CEO Jason Camm said: “The FDA's acceptance of our NDA submission for review is an important milestone, taking us another step closer to a new treatment option for children living with infantile-onset NPC. The FDA's granting of a priority review further reinforces the high unmet need in infantile-onset NPC.

“Our rigorous and comprehensive NDA package incorporated feedback from the FDA and the NPC community, and we remain focused on advancing our application. We are grateful to the patients, families, clinicians and advocates who made this submission possible.”