Intrabio announces FDA approval of their drug Levacetylleucine (Aqneursa) for the treatment of Niemann-Pick Disease Type C

On 25th September 2024, the pharmaceutical company Intrabio announced that the FDA in the US has approved their small molecule drug Levacetylleucine for the treatment of Niemann-Pick Disease Type C (NPC). NPC is a rare lysosomal storage disorder, in which ataxia can be a presenting symptom.

The FDA approval follows positive data from their phase III clinical trial which showed significant improvement in neurological symptoms and functional benefits in adults and children with NPC taking Levacetylleucine within 12 weeks of administration. 

We previously reported on the positive results from their multinational clinical trial testing levacetylleucine in NPC. Levacetylleucine resulted in a clinically meaningful improvement of symptoms, including in the Scale for the Assessment and Rating of Ataxia (SARA) score. The treatment was observed to be safe and well-tolerated, with no drug-related serious adverse events. The trial took place in the USA, European Union and the UK. A representative of Intrabio has previously attended Ataxia UK’s patient conference to present data on this trial. Intrabio has also in the past explored the potential of this drug in spinocerebellar ataxias, so it may offer hope in the future.  

Read the press release from Intrabio on their website here.