IntraBio has submitted a supplemental New Drug Application (sNDA) to the US Food and Drug Administration (FDA) for levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T), a rare, progressive, inherited neurodegenerative disorder.

This submission represents the first regulatory application submitted to the US Food and Drug Administration (FDA) seeking approval of a therapy for the treatment of Ataxia-Telangiectasia.

The sNDA seeks to expand the label of levacetylleucine, marketed as AQNEURSA, to include A-T. AQNEURSA is currently approved in the United States (US) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing at least 15 kg.

The submission is supported by data from a pivotal phase-III clinical trial evaluating levacetylleucine in adult and paediatric patients with A-T. In this randomised, double-blind, placebo-controlled, crossover study, levacetylleucine met its primary endpoint and key secondary endpoints with high statistical significance. Levacetylleucine was generally safe and well-tolerated, with no drug-related serious adverse events observed, consistent with its established safety profile.