24 September 2024 - Today, the US FDA approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C in adults and paediatric patients weighing at least 15 kg.

The safety and efficacy of Aqneursa for the treatment of Niemanm-Pick disease type c were evaluated in a randomised, double-blind, placebo controlled, two period, 24-week cross-over trial.

Read FDA press release