Prognia
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🇪🇺EU · EMAAuthorisedATC: N07XX27Orphan Medicine

Aqneursa(L-acetylleucine)

Niemann-Pick Disease, Type C

Marketing Authorisation Holder: Intrabio Ireland Limited

EMA Authorisation: 19/01/2026

INN: L-acetylleucine

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.

Official EMA Resources

View on EMA (ema.europa.eu)

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Medical disclaimer: This information is intended for qualified healthcare professionals. Always verify with the official EMA Summary of Product Characteristics (SmPC) before clinical use.