IntraBio’s AQNEURSA earns positive CHMP opinion, bringing Niemann-Pick disease type C therapy closer to EU approval

IntraBio Inc., a clinical-stage biopharmaceutical company, has secured a major regulatory milestone with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issuing a positive recommendation for AQNEURSA (levacetylleucine) to treat Niemann-Pick disease type C (NPC). The recommendation positions AQNEURSA as a potentially transformative therapy for the rare, neurodegenerative lysosomal storage disorder, and follows its U.S. FDA approval in September 2024. A final decision from the European Commission is anticipated later this year.

This development strengthens IntraBio’s position in the rare disease therapeutics market and brings the promise of a first-in-class, disease-modifying treatment to European patients suffering from NPC. The CHMP recommendation was grounded in data from a pivotal Phase III trial and a long-term extension study demonstrating significant symptomatic relief, sustained neurological benefits, and neuroprotective effects. AQNEURSA now stands to become a foundational therapy across the transatlantic NPC care ecosystem, especially in light of the limited treatment options available for this progressive, often fatal disease.

Why did AQNEURSA receive a positive CHMP recommendation?

The CHMP’s endorsement follows the results of IntraBio’s IB1001-301 Phase III clinical trial (NCT05163288), a randomized, placebo-controlled study involving 60 pediatric and adult patients diagnosed with NPC. Within 12 weeks of treatment, AQNEURSA met its primary and all secondary endpoints, demonstrating statistically significant improvements in neurological symptoms and patient functioning when compared to placebo. Improvements were particularly observed in motor coordination, swallowing, and speech—domains where NPC patients typically show irreversible decline.

Long-term extension data also contributed to the CHMP’s assessment. These findings, showing progressive improvement and stabilization of disease markers over time, support AQNEURSA’s potential disease-modifying effect. The durability of benefit and tolerability profile reinforced the case for regulatory advancement. Results from the study were published in the February 2024 issue of The New England Journal of Medicine, lending further scientific validation to the therapeutic’s impact.

According to Dr. Marc Patterson, Chief Medical Officer of IntraBio, the decision “reflects the strength of our clinical data and the potential for AQNEURSA to be a foundational therapy for NPC, delivering meaningful benefits for patients.” IntraBio anticipates AQNEURSA’s approval in the EU to pave the way for expanded access and broader adoption.

How AQNEURSA fits into the broader rare disease drug landscape

Niemann-Pick disease type C, while classified as ultra-rare, has drawn growing interest from drug developers due to its genetic complexity and high unmet medical need. Characterized by defective intracellular lipid trafficking, the disease causes progressive neurodegeneration, with onset occurring in both pediatric and adult populations. Symptoms range from ataxia and dysphagia to dementia and psychosis, leading to severe disability and premature death. Current treatment options are limited, with symptomatic management being the primary approach in Europe pending regulatory approval of AQNEURSA.

The global rare neurological disorder drug market, valued at approximately $7 billion in 2023, is projected to grow at a CAGR exceeding 7% through 2030, driven by regulatory incentives, orphan drug designations, and expanding genetic diagnostics. In this context, AQNEURSA’s CHMP milestone positions IntraBio as a key innovator with the potential to scale its development platform across other lysosomal storage disorders and ataxias.

IntraBio’s therapeutic platform targets neurological dysfunction across several rare diseases using small-molecule approaches. AQNEURSA, an orally administered, enantiomerically pure derivative of N-acetyl-DL-leucine, is designed to restore cerebellar function and neuronal homeostasis. The company has also reported promising exploratory results for the therapy in conditions such as GM2 gangliosidosis and ataxia-telangiectasia, reflecting a platform strategy for pipeline extension.

Patient and community response

The NPC patient community, long underserved by traditional pharma pipelines, has widely welcomed the CHMP opinion. Carmelo Fernández, President of Fundación Niemann-Pick de España, stated, “We have waited years for a therapy that can make a meaningful difference in the lives of people with NPC, and today’s announcement brings us one step closer.” Advocacy groups across Europe, including those in the UK, France, and Germany, have also echoed their support, emphasizing the potential of AQNEURSA to become the first disease-modifying treatment available to European patients.

The therapy’s approval in the United States under the FDA’s Accelerated Approval pathway in 2024 marked a pivotal moment for American patients and was accompanied by a post-marketing commitment from IntraBio to continue long-term follow-up studies. In Europe, the CHMP recommendation sets the stage for national health authorities and payers to evaluate reimbursement pathways, with some markets likely to adopt early access programs before full pricing negotiations are concluded.

Institutional and investor sentiment

Although IntraBio is privately held and not listed on public markets, the CHMP recommendation is likely to attract further institutional attention from rare disease-focused venture capital firms and strategic partners. IntraBio has previously raised over $80 million from global healthcare investors, and analysts expect the regulatory progress of AQNEURSA to catalyze additional licensing opportunities or regional commercialization partnerships.

The company may also benefit from EU orphan drug incentives, including market exclusivity, fee waivers, and scientific advice, which can substantially de-risk commercialization. With the CHMP nod following shortly after FDA approval, IntraBio is positioned for accelerated growth and broader investor engagement.

What’s next for AQNEURSA in Europe?

The European Commission (EC) will issue its final decision on AQNEURSA within 67 days of the CHMP opinion, following a standard review protocol. If approved, AQNEURSA will receive centralized marketing authorization, allowing commercial distribution across all 27 EU member states as well as Norway, Iceland, and Liechtenstein.

Commercial rollout strategies are expected to prioritize high-burden countries with established NPC diagnostic networks such as Germany, France, and the UK. IntraBio is reportedly preparing a phased launch supported by regional medical science liaisons and advocacy partnerships to drive disease awareness and treatment adoption.

Analysts anticipate the EC decision to act as a springboard for further regulatory filings in other global markets, including Japan, Canada, and Australia. AQNEURSA’s dual approval in the U.S. and EU could also prompt discussions around inclusion in newborn screening panels, as early diagnosis becomes more actionable with the availability of disease-modifying therapies.

How this aligns with long-term trends in EU rare disease policy

The CHMP’s backing of AQNEURSA aligns with the European Commission’s 2024-2029 rare disease action plan, which calls for accelerated development and approval of therapies for underserved populations. The plan supports coordinated EU-level efforts for clinical trial funding, patient registry development, and cross-border data sharing—areas where IntraBio could further engage through its expanding research footprint.

This regulatory milestone also reflects a broader shift in EMA’s approach to rare disease approvals, increasingly favoring robust functional outcomes and long-term patient benefit over traditional survival-based endpoints. AQNEURSA’s approval may therefore serve as a case study in adaptive trial designs and real-world data use for future ultra-rare therapy evaluations.

Analysts expect further platform growth from IntraBio

Beyond NPC, IntraBio is expected to leverage its AQNEURSA development data to expedite clinical development for other neurodegenerative orphan diseases. The company’s pipeline includes ongoing trials in ataxia-telangiectasia and GM2 gangliosidosis, both of which are characterized by overlapping lysosomal and cerebellar pathophysiology. Success with AQNEURSA may not only validate IntraBio’s approach but also lead to broader strategic collaborations with biotech or big pharma players seeking access to niche CNS markets.

Industry analysts believe IntraBio could become a leading player in the rare neurodegenerative space if it maintains regulatory momentum and executes effectively on market entry. The CHMP recommendation represents more than a milestone—it signals a shift in how neurological rare diseases may be managed globally over the next decade.