Single-dose treatment approved for sleeping sickness

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A single-dose oral treatment for sleeping sickness has been recommended for approval by the European Medicines Agency (EMA).

The decision could accelerate access to a simplified treatment for gambiense human African trypanosomiasis (HAT), the most common form of the disease in Central and West Africa.

The EMA’s committee for Medicinal Products for Human Use (CHMP) recommended approval of Acoziborole Winthrop as a one-day oral treatment for both early- and late-stage Trypanosoma brucei gambiense infection in adults and adolescents aged at least 12 years, and weighing over 40 kgs.

The recommendation was issued under the EU-M4all Pathway which allows the EMA to assess medicines intended for use outside the European Union, particularly for diseases with limited commercial markets. The assessment supports regulatory decisions in endemic countries such as the Democratic Republic of Congo and Guinea. It is a step toward updates to WHO treatment guidelines.

Human African Trypanosomiasis, sleeping sickness, is transmitted by tsetse fly and is almost always fatal if left untreated. Early symptoms include fever and headaches. In late-stage disease parasites invade the central nervous system, causing sleep disturbances, confusion, and seizures.

Acoziborole was developed by Sanofi, in partnership with DNDi (Drugs for Neglected Diseases Initiative). Earlier advances included the injectable NECT regimen and the first oral treatment, fexinidazole, which was approved for use in Africa in 2025, and requires a 10-day course. A single-dose therapy could simplify treatment delivery in remote settings.

The CHMP advice draws on Phase II/III trial data, published in The Lancet Infectious Diseases¹ which reported treatment success rates of up to 96% at 18 months in early and late stages of the disease.

WHO aims to eliminate gambiense sleeping sickness by 2030. Experts say a simple, field-friendly treatment could be critical as case numbers decline and surveillance becomes more challenging. Sanofi has pledged to donate the treatment through its philanthropic arm, Foundation S, ensuring free provision in endemic countries.

A study is now under way in the DRC and Guinea to evaluate the drug in children aged 1 to 14 years.

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