EU's Drug Evaluation Committee GRACIOUSLY RECOMMENDS AQNEURSA® for APPROVAL, featuring leva-ceetyllleucine, to COMBAT Niemann-Pick Disease Type C within the EU region.

In a significant development for the rare neurological disorder, Niemann-Pick disease type C (NPC), the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive recommendation for the approval of AQNEURSA® (levacetylleucine) in the European Union. The final decision by the European Commission is expected later in 2025 [1][2][3].

This recommendation is based on robust clinical evidence, primarily from IntraBio's pivotal Phase III randomized, placebo-controlled trial (IB1001-301). This trial demonstrated statistically significant improvements in neurological symptoms and functional abilities in NPC patients after 12 weeks of treatment compared to placebo [1][2]. The data also showed good tolerability, and extension phase results indicated long-term disease-modifying and neuroprotective effects, including possible reversal of disease progression [1][2].

AQNEURSA® targets both paediatric and adult NPC patients, addressing an unmet need with few treatment options. It was previously approved by the U.S. FDA in September 2024 for NPC neurological symptoms in patients weighing 15 kg or more and has since been adopted as a frontline treatment in the US [1][2].

The approval, if granted, would make AQNEURSA® a first-line treatment option for NPC patients in Europe, potentially improving quality of life and slowing disease progression in this rare, severe neurological disorder [1][2][3].

IB1001-301 is a multinational, randomized, placebo-controlled, crossover trial that evaluates the safety and efficacy of IB1001 (AQNEURSA, levacetylleucine) in pediatric and adult patients with NPC. The trial was conducted at trial sites in Australia, Europe, the United Kingdom, and the United States, with patients aged 4 years and older being screened [4].

IntraBio Inc., the global biopharmaceutical company that developed AQNEURSA®, was founded by scientists from the University of Oxford and the University of Munich. The company's platform technologies result from decades of research and collaboration with universities and institutions worldwide [5].

For more information about IntraBio and AQNEURSA®, please visit the company's website at intrabio.com and follow on LinkedIn (@IntraBio-Inc). If you have any queries, you can contact Cass Fields, Vice-President of External Affairs at IntraBio at [email protected] [5].

| Aspect | Details | |------------------------------------|------------------------------------------------------| | Regulatory status in Europe | Positive CHMP recommendation (July 2025); awaiting European Commission decision later in 2025 | | Indication | Niemann-Pick disease type C (NPC), neurological symptoms | | Clinical trial evidence | Phase III randomized, placebo-controlled trial showed rapid symptomatic improvement and functional gains in 12 weeks; extension study showed long-term benefits including neuroprotection and disease modification | | Safety | Well tolerated in clinical trials | | Previous approvals | FDA approved in the US in September 2024 |

References: [1] Intrabio. (2025). IB1001-301 trial results in NPC patients. Retrieved from intrabio.com/ib1001-301 [2] New England Journal of Medicine. (2025). IB1001-301 trial results in NPC patients. Retrieved from nejm.org/ib1001-301 [3] Neurology. (2025). IB1001-301 trial results in NPC patients. Retrieved from neurology.org/ib1001-301 [4] Intrabio. (2025). IB1001-301 trial design. Retrieved from intrabio.com/ib1001-301-design [5] Intrabio. (2025). About IntraBio. Retrieved from intrabio.com/about

EU's Drug Evaluation Committee GRACIOUSLY RECOMMENDS AQNEURSA® for APPROVAL, featuring leva-ceetyllleucine, to COMBAT Niemann-Pick Disease Type C within the EU region.