FDA Extends Review of Beren’s NPC Thearpy

Rare Daily Staff

The U.S. Food and Drug Administration has extended its review of Beren Therapeutics’ New Drug Application for adrabetadex, a potential treatment for infantile-onset Niemann-Pick disease type C, pushing the decision date to November 17, 2026.

The three-month extension follows the FDA’s classification of the company’s March 2026 submission as a major amendment, after Beren provided additional information and clarifications in response to an agency request. Under FDA guidelines, such amendments reset the review clock to allow sufficient time for evaluation.

The delay highlights the FDA’s continued scrutiny of therapies for ultra-rare diseases, particularly those involving complex delivery methods such as intrathecal administration.

Infantile-onset Niemann-Pick disease type C is a progressive neurodegenerative disorder caused by mutations in the NPC1 or NPC2 genes, leading to the accumulation of cholesterol within cells. Patients who develop neurological symptoms early in life face particularly poor outcomes, with average survival of just over five years in the most severe cases.

Adrabetadex, a cyclodextrin-based therapy administered intrathecally, is designed to address the underlying defect in Niemann-Pick disease type C by restoring intracellular cholesterol trafficking. The investigational therapy has received Priority Review, as well as Orphan Drug and Breakthrough Therapy designations, underscoring the high unmet need in this rare pediatric disorder.

Clinical and nonclinical data to date suggest that adrabetadex is generally well tolerated, with a safety profile established over more than a decade of development. Reported adverse events include hearing impairment, which can be managed with hearing aids, as well as fatigue and ataxia following dosing.

Beren CEO Jason Camm said the company will continue working closely with regulators to advance the application, emphasizing the urgency for affected families. The company is also maintaining access to adrabetadex through an Expanded Access Program for eligible patients.