The US Food and Drug Administration (FDA) announced on Tuesday that it has approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and paediatric patients weighing at least 15 kilograms.

This approval was granted to UK-based pharmaceutical company IntraBio Inc.

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction.

The safety and efficacy of Aqneursa were assessed in a randomised, double-blind, placebo-controlled, two-period, 24-week crossover study involving 60 subjects. To qualify for the study, patients had to be four years of age or older with a confirmed diagnosis of NPC and at least mild disease-related neurological symptoms. Participants could receive miglustat, an enzyme inhibitor, as background treatment in the study.

The primary efficacy outcome was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). The fSARA consists of the gait, sitting, stance and speech disturbance domains of the original SARA with modifications to the scoring responses. On average, participants treated with Aqneursa for 12 weeks showed a better outcome in the fSARA score compared to when they were treated with placebo.