Beren Therapeutics P.B.C., the parent company of Mandos LLC, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to adrabetadex, an investigational therapy for infantile-onset Niemann-Pick disease type C (NPC). The designation is intended to accelerate the development and review of promising treatments for serious or life-threatening conditions.
The FDA’s decision was based on an externally controlled survival analysis that indicated adrabetadex may significantly improve survival outcomes for patients with infantile-onset NPC, a rare and rapidly progressive neurodegenerative disorder. The analysis was evaluated alongside supportive biomarker and nonclinical data, collectively demonstrating the therapy’s potential to address an unmet medical need.
Adrabetadex had previously received BTD in 2016 under a former sponsor, but the designation was later rescinded due to results from a 12-month Phase 2b/3 clinical trial. Beren reacquired the program in 2021 from Mallinckrodt and has since worked closely with the FDA, clinicians, and the patient community to strengthen the data package supporting the drug’s development. This effort ultimately contributed to the reinstatement of the Breakthrough designation.
Jason Camm, CEO of Beren Therapeutics, called the new designation a significant milestone for families affected by infantile-onset NPC. He expressed gratitude to patients, caregivers, clinicians, and advocates who have supported the program over the years. “We are grateful to the people living with NPC and their caregivers, clinicians, and advocates who have worked with us through a long development path,” he said.
Dr. Elizabeth Berry-Kravis, Professor of Pediatrics at Rush University Medical Center and principal investigator for the expanded access program (EAP), emphasized the potential impact of the survival findings. “Infantile-onset NPC is a devastating, rapidly fatal diagnosis. Seeing statistically significant improvements in survival signals a meaningful shift in what is achievable for these patients,” she noted.
In addition to BTD, adrabetadex holds Orphan Drug and Rare Pediatric Disease designations, underscoring the therapy’s potential benefit in treating a critically underserved population. Beren Therapeutics plans to submit a New Drug Application (NDA) in the near future and anticipates that adrabetadex will qualify for Priority Review once filed.
The renewed designation marks a critical step forward in bringing a potentially life-extending treatment closer to patients affected by one of the most severe forms of NPC.