THOUSAND OAKS, Calif. — 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.
“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 at: https://www.BerenTx.com or visit Beren’s LinkedIn channel.
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