Aqneursa (L-acetylleucine)

OrphanAdditional monitoring

Region: 🇪🇺 EU
Active substance: L-acetylleucine
Company: Intrabio Ireland Limited
Status: Authorised
Therapeutic area: Niemann-Pick Disease, Type C
CHMP opinion: Thu 13 Nov 2025
EU approval: Mon 19 Jan 2026
Designations: Orphan, Additional monitoring
Application: EMEA/H/C/006327
ATC: N07XX27
Official record: www.ema.europa.eu

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.

Regulatory milestones

Mon 19 Jan 2026🇪🇺 EU approval (EC decision)

Aqneursa (L-acetylleucine) approved in the EU

Marketing authorisation to Intrabio Ireland Limited; Orphan, Additional monitoring · source

Thu 13 Nov 2025🇪🇺 CHMP positive opinion

Aqneursa (L-acetylleucine): CHMP positive opinion

Intrabio Ireland Limited; Orphan, Additional monitoring · source

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