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 placebo-controlled 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, including on the Scale for the Assessment and Rating of Ataxia (SARA) and Spinocerebellar Ataxia Functional Index (SCAFI).
IntraBio’s Chief Deveopment Officer Taylor Fields commented,
“IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC. We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that Levacetylleucine may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop Levacetylleucine for these additional indications.”
We previously reported on the positive results from IntrabBio’s multinational Phase IIb clinical trials testing levacetylleucine in NPC and GM2 Gangliosidosis. 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 multiple inherited cerebellar ataxias, including Ataxia-Telangiectasia, Spinocerebellar ataxias and episodic ataxias, so it may offer hope in the future. Read the press release from IntraBio on their website here.
Read the press release from Intrabio on their website here.
