July 30 2025: The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending AQNEURSA® (levacetylleucine) for approval in the EU for the treatment of Niemann-Pick disease type C (NPC). This recommendation positions AQNEURSA as a potential first-line therapy for a disease with limited treatment options and severe neurological decline in both paediatric and adult patients. A final decision by the European Commission is expected later in 2025.
The CHMP recommendation is based on IntraBio’s pivotal Phase III randomised, placebo-controlled clinical trial (IB1001-301; NCT05163288), which demonstrated statistically significant improvements in neurological symptoms and functional abilities within 12 weeks of treatment compared to placebo, across all primary and secondary endpoints. AQNEURSA was also well tolerated throughout the study.¹ These findings were complemented by extension-phase data showing long-term disease-modifying and neuroprotective effects, including reversal of disease progression.²
AQNEURSA previously received FDA approval in the United States in September 2024 for the treatment of neurological symptoms of NPC in patients weighing ≥15 kg.⁵ Following this, it has been rapidly adopted as a first-line treatment in the US.
NPC is a rare, autosomal recessive lysosomal storage disorder affecting approximately 1 in 100,000 live births, characterised by cerebellar ataxia, cognitive decline, motor dysfunction, and multi-system involvement.³⁴⁶⁷ The condition is progressive and fatal, significantly impairing quality of life.
IntraBio is also advancing its research pipeline, having completed recruitment for a Phase III trial evaluating levacetylleucine in Ataxia-Telangiectasia, with a data readout expected in Q1 2026.
References:
- Bremova-Ertl T, et al. N Engl J Med. 2024;390:421–431
- Patterson MC, et al. Neurology. 2025;105(1):e213589
- Geberhiwot T, et al. Orphanet J Rare Dis. 2018;13:50
- Patterson MC, et al. Orphanet J Rare Dis. 2013;8:12; NORD. NPC Signs & Symptoms. 2023
- AQNEURSA. Prescribing Information. IntraBio Inc
- Burton BK, et al. Mol Genet Metab. 2021;134:182–187
- Vanier MT. Orphanet J Rare Dis. 2010;5:16
