EMA — authorised 19 January 2026
- Application: EMEA/H/C/006327
- Marketing authorisation holder: Intrabio Ireland Limited
- Local brand name: Aqneursa
- Indication: 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.
- Pathway: orphan
- Status: approved
The European Medicines Agency (EMA) has granted marketing authorisation for Aqneursa, a medicinal product developed by Intrabio Ireland Limited. Aqneursa is indicated for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease. It can be used in combination with miglustat or as a monotherapy in patients where miglustat is not tolerated. The approval was granted under the orphan designation, which is reserved for medicines that treat rare diseases. Aqneursa has been approved for use in adults and children aged 6 years and older who weigh at least 20 kg.