Aqneursa
What Aqneursa is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
Key facts
- Brand
- Aqneursa
- Active substance
- L-acetylleucine
- Disease area
- Niemann-Pick Disease, Type C
- Medicine group
- Other nervous system drugs
- ATC code
- N07XX27
- Marketing authorisation holder
- Intrabio Ireland Limited
- Authorised in the EU since
- 2026
Official information
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Aqneursa used for?
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.
What is the active substance in Aqneursa?
Aqneursa contains L-acetylleucine.
Who makes Aqneursa?
The EU marketing authorisation holder is Intrabio Ireland Limited.
Is Aqneursa an orphan medicine?
Yes. Aqneursa is designated an orphan medicine in the EU, for a rare disease (Niemann-Pick Disease, Type C).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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