• 27 Jan 2026
• Admin
• News Article

European Commission Approves IntraBio’s Aqneursa for Niemann-Pick Type C

IntraBio Inc has received European Commission marketing authorization for Aqneursa (levacetylleucine) to treat neurological manifestations of Niemann-Pick Type C (NPC) disease.

The approval follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency.

Aqneursa is now approved in the European Union for:

• Adults and children aged 6 years and older
• Patients weighing at least 20 kg
• Use in combination with miglustat, or as monotherapy when miglustat is not tolerated

A milestone for the NPC community

“This approval represents a significant milestone for the Niemann-Pick Type C community in Europe,” said Mallory Factor, CEO of IntraBio.

She added that the decision reflects years of scientific work and collaboration with clinicians and patient organizations, and marks an important step toward expanding access to treatment.

Phase III data supporting approval

The authorization is based on a phase III randomized, double-blind, placebo-controlled, crossover study in patients with NPC.

Key findings:

• Statistically significant and clinically meaningful improvement in neurological signs and functioning after 12 weeks
• Improvement measured using the Scale for the Assessment and Rating of Ataxia (SARA)
• Benefits maintained in the ongoing open-label extension phase

Observational comparisons with a natural history cohort showed:

• 118% reduction in annual disease progression after one year
• Similar reduction after two years
• Measured using the 5-domain NPC Clinical Severity Scale (NPC-CSS)

Expert perspective

Professor Kyriakos Martakis, associate professor in paediatrics at Justus Liebig University Giessen and principal investigator, said:

“Niemann-Pick disease Type C is a rare and relentlessly progressive neurological disorder. The availability of an approved treatment addressing neurological manifestations represents an important development for clinicians and, most importantly, for affected individuals across Europe.”

About Aqneursa (levacetylleucine)

• First-in-class modified amino acid
• Designed to correct metabolic dysfunction and improve cellular energy production
• Delivered to all tissues, including the central nervous system
• Available as 1 g granules for oral suspension
• Orphan medicinal product designation during development

The most commonly reported adverse reaction is flatulence. Aqneursa is also approved in the United States for adults and paediatric patients weighing ≥15 kg with NPC.

About Niemann-Pick Type C

Niemann-Pick Type C is a rare, autosomal recessive lysosomal storage disorder affecting approximately 1 in 100,000 live births.

The disease causes:

• Progressive neurological deterioration
• Psychiatric and systemic symptoms
• Cerebellar ataxia and organ dysfunction
• Substantial reduction in quality of life

IntraBio advances broader neurodegenerative pipeline

IntraBio also announced positive results from a phase III study in Ataxia-Telangiectasia, a neurodegenerative disease with no approved treatments. The company plans to advance regulatory submissions to:

• US FDA
• European Medicines Agency

Additional global regulatory authorities