FDA Grants Breakthrough Therapy Designation to Adrabetadex for Infantile-Onset NPC

This FDA designation is reserved for medicines that treat serious or life-threatening conditions and show substantial potential to improve upon existing therapies. It helps to accelerate the development and review process. For adrabetadex, the designation is supported by survival data from treated patients compared with untreated individuals.

Adrabetadex (also known as VTS-270) is a specially formulated mixture of 2-hydroxypropyl-β-cyclodextrin isomers developed by Mandos, a subsidiary of Beren Therapeutics P.B.C. It aims to correct the underlying problem in NPC by restoring normal cholesterol transport within cells. The treatment has shown promise in extending survival for children with infantile-onset NPC (symptoms appearing between birth and six years of age). It has already received Orphan Drug and Rare Paediatric Disease designations in the United States, and the company intends to submit a New Drug Application (NDA) shortly.

The treatment has generally been well tolerated, with common side effects including hearing impairment (often managed with hearing aids), fatigue, and temporary ataxia after dosing. Adrabetadex is not yet approved by any regulatory authority, including the MHRA in the UK, but this latest designation is an important milestone.

NPC is a rare, inherited neurodegenerative disorder that affects approximately 1 in 100,000–150,000 births. Mutations in the NPC1 or NPC2 genes impair the body’s ability to transport cholesterol and other lipids within cells, leading to their accumulation in organs such as the brain, liver, and spleen. Infantile-onset NPC is particularly severe, with early symptoms including developmental delay, seizures, and progressive loss of motor skills, often resulting in a significantly shortened life expectancy. Advances such as this offer real hope to families facing this devastating diagnosis.

Jason Camm, CEO of Beren Therapeutics, said: “This newly granted designation represents an important milestone in the evaluation of adrabetadex for people living with infantile-onset NPC. 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.”

Dr Elizabeth Berry-Kravis, a leading expert and principal investigator, added: “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.”

This FDA recognition could help speed up access to a potential new treatment for the youngest and most severely affected NPC patients. Although the designation is from the United States, it may influence global research and regulatory discussions. NPUK continues to advocate for better therapies and support services here in the UK.

If you or someone you know is affected by NPC, please contact NPUK for advice and support: info@npuk.org or 0191 415 0693.