Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann Pick Disease Type C

Treatment for: Niemann-Pick Disease

Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann Pick Disease Type C

THOUSAND OAKS, Calif. – February 23, 2026 – Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for adrabetadex, an investigational cyclodextrin therapy designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C (NPC). If approved, adrabetadex would represent a first-in-class, disease-modifying approach to treat infantile-onset NPC, a rare and rapidly fatal pediatric neurodegenerative disorder. The FDA assigned adrabetadex a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026.

Accepted for priority review with Prescription Drug User Fee Act (PDUFA) action date of August 17, 2026

If approved, adrabetadex would be the only therapy to directly target the underlying pathophysiology of Niemann-Pick disease type C (NPC)

Comprehensive NDA application includes data showing long-term survival benefit, slowed disease progression, confirmatory biomarkers and nonclinical evidence

"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," said Jason Camm, Chief Executive Officer of Beren Therapeutics P.B.C. "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."

The NDA for adrabetadex is supported by data demonstrating a clinically meaningful survival benefit in infantile-onset patients. This evidence is from an externally controlled analysis intended to serve as a single adequate and well-controlled study. The comprehensive submission also includes data showing slowed disease progression, confirmatory biomarker and nonclinical findings and patient experience narratives.

Key findings were recently presented at WORLDSymposium 2026, the largest annual scientific meeting in the field of lysosomal storage disease, including survival data in infantile-onset NPC: a 71% reduction in the risk of mortality in adrabetadex-treated patients compared with matched external controls (HR 0.289; 95% CI, 0.141-0.593; P < 0.0001).

"Infantile-onset NPC is the fastest progressing subtype and has limited treatment options," said Elizabeth Berry-Kravis, M.D., Ph.D., professor of Pediatrics at Rush University Medical Center and principal investigator of the Expanded Access Program. "The approval of adrabetadex would mark the first time we have a disease-modifying therapeutic option, which is urgently needed for these young children and their families."

Beren previously announced that in 2025 the FDA granted adrabetadex Breakthrough Therapy Designation, a status that accelerates the development of drugs for serious or life-threatening conditions when early evidence suggests a substantial improvement over existing therapy.

About Niemann-Pick Disease Type C

Niemann-Pick disease type C (NPC) is a rare, autosomal-recessive, severe, neurodegenerative disorder caused by pathologic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired cholesterol trafficking resulting in progressive neurological decline and premature death. Infantile-onset NPC refers to NPC in infants and children who first experience neurological symptoms between 0 and 6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean survival of ~5.6 years for early infantile-onset (age of neurological onset <2 years) and ~13.4 years for late-infantile onset (2 to <6 years).

About Adrabetadex

Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation as a treatment for Niemann-Pick disease type C (NPC). The data suggest that by re-establishing intracellular cholesterol trafficking, adrabetadex is designed to directly address the underlying pathology of NPC. Data from clinical trials and expanded access programs suggest that adrabetadex is generally well tolerated. The main adverse events associated with adrabetadex include hearing impairment that can be managed with hearing aids when necessary, and post-dose fatigue and/or ataxia. Adrabetadex has not been approved by the FDA or any other health authority at this time.

About Beren Therapeutics P.B.C.

Beren Therapeutics P.B.C. is a founder-led, clinical-stage biotechnology company pioneering the discovery, development and commercialization of cyclodextrin-based therapeutics for conditions characterized by defective cholesterol trafficking. Beren and its subsidiary Mandos LLC are committed to the development of adrabetadex for individuals living with Niemann-Pick disease type C (NPC) and have supported the NPC community by providing access to adrabetadex through an Expanded Access Program (EAP).

Beren's public benefit purpose is to discover, develop and deliver novel therapies that provide optimal benefit for patients, and to do so by integrating the needs of patients, caregivers, clinicians and health systems from the beginning of the development process and maintaining a long-term focus on delivering meaningful therapies and access.

Beren is headquartered in Thousand Oaks, Calif. To learn more about Beren, the adrabetadex program and Beren's cholesterol-trafficking focused therapeutic strategy, visit the company's newly launched website or visit Beren's LinkedIn channel.

Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to, statements regarding the FDA's review of the NDA for adrabetadex; the timing and outcome of regulatory review; the potential for adrabetadex to provide clinical benefit or to address the underlying pathology of Niemann-Pick disease type C; the potential for approval or commercialization; the ability to continue providing access through expanded access; and Beren's plans to expand its pipeline. Actual results may differ materially due to risks and uncertainties including those associated with clinical development, regulatory review, manufacturing, safety and efficacy outcomes and other factors.

Source: Beren Therapeutics P.B.C.

Source: HealthDay

Beren, Through its Subsidiary Mandos, Submits New Drug Application to U.S. FDA for Adrabetadex in Infantile-Onset NPC - December 19, 2025

Adrabetadex FDA Approval History

More news resources

FDA Medwatch Drug Alerts

Daily MedNews

News for Health Professionals

New Drug Approvals

New Drug Applications

Drug Shortages

Clinical Trial Results

Generic Drug Approvals

Drugs.com Podcast

Subscribe to our newsletter

Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Posted : 2026-02-25 13:58

Disclaimer

Every effort has been made to ensure that the information provided by Drugslib.com is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Drugslib.com information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Drugslib.com does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Drugslib.com's drug information does not endorse drugs, diagnose patients or recommend therapy. Drugslib.com's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners.

The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Drugslib.com does not assume any responsibility for any aspect of healthcare administered with the aid of information Drugslib.com provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann Pick Disease Type C