Department of Pharmacology research leads to FDA approval for new NPC treatment — University of Oxford, Medical Sciences Division

By Rekha Reddipalli on February 6, 2025

IntraBio, co-founded by Department of Pharmacology academics Professors Fran Platt, Antony Galione and Grant Churchill, ran a successful international clinical trial for a new drug Aqneursa which showed significantly improved neurological outcomes over a 12 week period for a cohort of 60 patients, aged four years and over, with a confirmed diagnosis of NPC compared to a control group. Despite the recent proliferation of Drug Discovery Units in many Universities, it is nevertheless still rare for academics to take a new molecular entity from pre-clinical to phase III clinical trials culminating in regulatory approval and marketing for patients. The first prescriptions for Aqneursa have already been written.

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by changes in either the NPC1 or NPC2 gene, affecting the transport of cholesterol and other lipids out of the lysosome. As a result, cells do not function as they should, ultimately causing organ damage. On average, individuals affected by this devastating disease only live for about 13 years. The new drug, Aqneursa, is only the third drug approved for the treatment of NPC, including miglustat, which was the first drug to be approved for lysosomal storage diseases that was developed by the Platt laboratory.

Read the full story on the Department of Pharmacology website.

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