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EMA approval of IntraBio’s Aqneursa marks a major milestone for the Department of Pharmacology, with further Phase 3 success in Ataxia Telangiectasia

On 21 January, the European Medicines Agency (EMA) approved Aqneursa, a new treatment for Niemann-Pick Disease Type C (NPC) in adults and children, marking a major milestone for research originating in the Department of Pharmacology. Aqneursa already received US FDA approval (September 2024), enabling patient access on both sides of the Atlantic.

Professor Fran Platt Aqneursa was developed by IntraBio, a start-up co-founded by Department of Pharmacology academics Professors Fran Platt, Antony Galione and Grant Churchill. The approval follows a successful international Phase 3 clinical trial showing significantly improved neurological outcomes over 12 weeks in 60 patients aged four years and older with confirmed NPC. Academic-led programmes progressing from pre-clinical research through to regulatory approval remain rare, highlighting the significance of this achievement.

In addition to the EMA approval, IntraBio has also recently announced positive Phase 3 results in another rare neurodegenerative disorder, Ataxia Telangiectasia, underlining the broader potential of the company’s therapeutic approach for ataxias and other neurological diseases.

Professor Fran Platt, Head of the Department of Pharmacology, said: “The EMA approval of Aqneursa is a tremendous milestone and a powerful example of how academic research can deliver life-changing treatments for patients with rare diseases.”

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