New PDUFA target action date of November 17, 2026 allows FDA additional time to complete its review
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and a leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, announced that the U.S. Food and Drug Administration (FDA) has extended by three months the review timeline of its New Drug Application (NDA) for adrabetadex for the treatment of infantile-onset Niemann-Pick disease, type C (I-NPC). The new Prescription Drug User Fee Act (PDUFA) target action date is November 17, 2026.
The extension follows Beren’s March 18, 2026 response to an FDA information request that included updates and clarifications to existing data and supporting documentation. The FDA classified the response as a Major Amendment to the NDA, which under FDA regulations extends the review period by three months.
“We are committed to working with the Agency, physicians, and NPC families to deliver this critical therapy for patients living with infantile-onset NPC as quickly as possible,” said Jason Camm, Chief Executive Officer of Beren Therapeutics P.B.C.
Beren remains committed to providing adrabetadex to eligible patients with NPC through an ongoing Expanded Access Program (EAP).
The NDA for adrabetadex was granted Priority Review by the FDA, and adrabetadex previously received Orphan Drug Designation and Breakthrough Therapy Designation.
About Infantile-Onset Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a rare, autosomal-recessive, severe, heterogeneous, neurodegenerative disorder caused by pathogenic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired intracellular cholesterol trafficking resulting in progressive neurological decline and premature mortality. Infantile-onset NPC (I-NPC) refers to NPC in infants and children who first experience neurological symptoms <6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean ages of death 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 suitable for intrathecal delivery, under investigation as a treatment for Niemann-Pick disease, type C (NPC). Clinical and nonclinical data demonstrate that adrabetadex directly targets the underlying pathophysiology of NPC by re-establishing intracellular cholesterol trafficking. Adrabetadex is generally well tolerated, with a well-characterized safety profile established over more than a decade of clinical development. The most common adverse events are hearing impairment (manageable with hearing aids when necessary) and post-dose fatigue and 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 website or visit Beren’s LinkedIn channel.
Contacts
Media Contact:
Beren Therapeutics Media Relations
media@berentx.com
