Is Ambroxol legal in the United Kingdom?

An over-the-counter cough medicine in much of the world that raises glucocerebrosidase levels in the brain at high doses. The one randomised placebo-controlled trial in Parkinson disease dementia, 55 patients over 52 weeks, hit target engagement and missed both primary clinical endpoints.

Ambroxol has been sold as a mucolytic for decades. Its second life began in 2009 when a screen identified it as an enzyme enhancement agent for Gaucher disease, meaning it acts as a pharmacological chaperone that helps the enzyme glucocerebrosidase fold and reach the lysosome.

Mechanism: A pharmacological chaperone for glucocerebrosidase. It binds the enzyme in the endoplasmic reticulum at neutral pH, stabilising the folded form so more of it traffics to the lysosome, and releases it at the acidic lysosomal pH.

Regulatory history: Not approved in the United States for any indication. Widely available over the counter as a mucolytic in Europe, Asia and Latin America. The Parkinson disease work is investigational everywhere and uses doses far above the mucolytic range.

Licenses medicines for the UK market. A compound without a marketing authorisation is an unlicensed medicine, which can only lawfully be supplied for human use through a prescriber under the specials scheme, not sold as a research product.

The classification above is the The Longevity Archive reading of Medicines and Healthcare products Regulatory Agency listings for Ambroxol at the reviewed date, not a legal opinion. Rules change, and importing, prescribing and possessing a compound can each be treated differently. Check the regulator directly before acting on anything here.