IntraBio has submitted the variation application to the European Medicines Agency (EMA) to expand the approved Marketing Authorisation Application (MAA) for AQNEURSA (levacetylleucine) to include the treatment of Ataxia-Telangiectasia (A-T).

This submission represents the first regulatory application submitted to the EMA seeking approval of a therapy for the treatment of A-T, a rare, progressive, inherited neurodegenerative disorder. The variation application is subject to EMA validation before the start of formal scientific review.

In the US, IntraBio’s supplemental New Drug Application (sNDA) for AQNEURSA for A-T has been accepted for review by the US Food and Drug Administration (FDA) and granted Priority Review (PR), with a Prescription Drug User Fee Act (PDUFA) target action date of September 19, 2026.

The variation application seeks to expand the label of AQNEURSA to include A-T. AQNEURSA is currently approved in the European Economic Area for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat, and in the US for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.