The FDA has extended its review of Beren Therapeutics’ adrabetadex NDA for infantile-onset Niemann-Pick disease type C, setting a new PDUFA date of November 17, 2026.
Written By: Disha Jadhav, BPharm
Reviewed By: Pharmacally Editorial Team
Beren Therapeutics P.B.C. announced that the U.S. Food and Drug Administration (FDA) has extended its review of the New Drug Application (NDA) for adrabetadex, an investigational therapy for infantile-onset Niemann-Pick disease type C (I-NPC).
The agency has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2026, following classification of Beren’s March 18 submission as a Major Amendment. Under FDA regulations, such amendments extend the review period by up to three months, and in this case the full extension was applied.
Regulatory Context
Adrabetadex is under Priority Review and has previously received Breakthrough Therapy and Orphan Drug Designations, reflecting the significant unmet need in NPC.
The company’s March submission provided updates and clarifications to existing data and supporting documentation, prompting the FDA’s extension decision.
Major Amendments do not necessarily indicate a regulatory setback but reflect the FDA’s need for additional time to review newly submitted information before completing its assessment.
Disease Background
Niemann-Pick disease type C is a rare, inherited lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes, leading to impaired intracellular cholesterol trafficking. Infantile-onset NPC, defined by neurological symptom onset before six years of age, is associated with rapid disease progression, severe neurological decline, and poor survival outcomes.
Mechanism and Development
Adrabetadex is a proprietary intrathecal formulation of 2-hydroxypropyl-β-cyclodextrin isomers that restores disrupted intracellular cholesterol transport. The therapy has been evaluated across multiple Phase 1–3 studies in both pediatric and adult NPC populations, along with long-term follow-up programs spanning more than a decade.
Clinical and nonclinical findings have shown that adrabetadex directly addresses the underlying disease mechanism in NPC. Reported adverse events include hearing impairment, which is often manageable with hearing aids, along with post-dose fatigue and ataxia.
Patient Access and Outlook
Chief Executive Officer Jason Camm said the company remains committed to working closely with regulators, physicians, and patient communities to bring the therapy to patients with infantile-onset NPC as quickly as possible.
While regulatory review continues, Beren will maintain access to adrabetadex through its U.S. Expanded Access Program for eligible NPC patients. If approved, adrabetadex could become the first therapy approved to directly target the underlying cholesterol trafficking defect that drives NPC pathology, representing a significant advance for patients affected by this rare and progressive disease.
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About the Writer
Disha Sanjay Jadhav (LinkedIn) is a pharmacy graduate and healthcare writer with a strong interest in clinical documentation and simplifying healthcare information for better reader understanding. She is enthusiastic, adaptable, and eager to take on new challenges while contributing to clear, accurate, and engaging medical and pharmaceutical content.