FDA Grants Breakthrough Therapy Designation to Adrabetadex for Infantile Onset Niemann Pick Type C
Beren Therapeutics P.B.C., parent company of Mandos LLC, has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for adrabetadex, an investigational drug for infantile-onset Niemann-Pick disease type C (NPC).
The designation is intended to accelerate the development of treatments for serious or life-threatening conditions where early clinical evidence suggests a substantial improvement over existing therapies.
The FDA’s decision was informed by an externally controlled survival analysis showing that adrabetadex improves survival in patients with infantile-onset NPC, supported by biomarker and nonclinical data.
Beren acquired the adrabetadex programme in 2021 and has since worked closely with the FDA and the NPC community to advance its development. Adrabetadex previously received BTD in 2016, but the designation was later rescinded following Phase 2b/3 data.
Adrabetadex also holds Orphan Drug and Rare Pediatric Disease designations. Beren plans to submit a New Drug Application (NDA) soon, with the therapy expected to be eligible for Priority Review.
Niemann-Pick disease type C is a rare, severe neurodegenerative disorder caused by mutations in NPC1 or NPC2, leading to cholesterol trafficking defects and progressive neurological decline.
Infantile-onset NPC occurs in children aged 0–6 years, with earlier onset associated with faster disease progression and lower survival.
Adrabetadex works by restoring intracellular cholesterol trafficking, addressing the underlying disease mechanism, and is generally well tolerated.
