Aqneursa

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Opinion

EMA has issued an opinion on this medicine

L-acetylleucine
Medicine Human Opinion
  • Application under evaluation
  • CHMP opinion
  • European Commission decision

Overview

Update as of 31 July 2025
The applicant for Aqneursa has requested a re-examination of EMA’s July 2025 opinion. Upon receipt of the grounds of the request, the Agency will re-examine its opinion and issue a final recommendation.

On 24 July 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Aqneursa1, intended for the treatment of neurological manifestations of Niemann-Pick Type C (NPC) disease in adults and children from 6 years of age weighing at least 20 kg.

The applicant for this medicinal product is IntraBio Ireland Ltd.

Aqneursa will be available as 1 g granules for oral suspension. The active substance of Aqneursa is levacetylleucine, a modified form of the amino acid leucine (ATC code: ‘not yet assigned’) that targets underlying processes of neurological dysfunction. While the mechanism of action of levacetylleucine is not yet fully understood, non-clinical studies have demonstrated that it corrects energy metabolism, including improved production of adenosine triphosphate, the main source of energy for cerebellar tissues and cells.

The benefits of Aqneursa are an improvement in neurological signs, symptoms and functioning in patients with NPC disease after 12 weeks of treatment, as measured using the scale for the assessment and rating of ataxia (SARA), compared with placebo, as observed in a randomised, double-blind, placebo-controlled, 2-period crossover phase 3 study.

The most common side effect with Aqneursa is flatulence.

The full indication is:

Aqneursa is indicated for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease, in combination with miglustat, or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.


1 This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained

Product details

Name of medicine
Aqneursa
Active substance
L-acetylleucine
International non-proprietary name (INN) or common name
L-acetylleucine
Therapeutic area (MeSH)
Niemann-Pick Disease, Type C
EMA product number
EMEA/H/C/006327

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Marketing authorisation applicant
Intrabio Ireland Limited
Opinion adopted
24/07/2025
Opinion status
Positive
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