In a milestone for global health research, the European Medicines Agency (EMA) has issued a positive scientific opinion for acoziborole, a single-dose oral treatment for gambiense human African trypanosomiasis—better known as sleeping sickness.
The disease, caused by the parasite Trypanosoma brucei gambiense and transmitted by the tsetse fly, has long plagued rural communities across sub-Saharan Africa. Developed through a partnership involving the Drugs for Neglected Diseases initiative and Sanofi, and supported by the European and Developing Countries Clinical Trials Partnership, the drug represents a rare convergence of scientific innovation and needs-driven research.
For more than a century, treatment for sleeping sickness has been defined by risk and complexity. Older therapies—some derived from arsenic—were toxic, difficult to administer, and required hospitalisation and invasive diagnostic procedures such as lumbar punctures. Acoziborole changes that paradigm.
Taken as a single oral dose, it has the potential to eliminate the need for spinal taps and prolonged hospital stays, enabling a simple “test-and-treat” strategy that can be delivered in remote clinics. For health systems with limited infrastructure, this shift could be transformative, bringing care closer to patients who have historically been hardest to reach.
The journey to this point has taken more than a decade of coordinated effort. EDCTP funding, combined with contributions from endemic countries and international partners, helped generate the clinical evidence needed for regulatory review. “This partnership has pooled resources to develop an effective treatment for a highly neglected disease,” said Maria Pilar Aguar Fernandez, chair of the Global Health EDCTP3 governing board. The collaboration has also helped ensure that the treatment can move beyond approval toward real-world use, including among children and underserved populations.
Two major initiatives now aim to translate clinical success into population-level impact. The ACOZI-KIDS project is working to extend the use of acoziborole to children aged 1 to 14, a group often overlooked in drug development. Clinical trials in the Democratic Republic of the Congo and Guinea completed recruitment in 2025, marking a critical step toward inclusive treatment access. Meanwhile, the STROGHAT consortium, led by the Institute of Tropical Medicine Antwerp, is testing a community-based screening and treatment model designed to halt transmission altogether.
Early results are striking. In just two years, STROGHAT screened more than 450,000 people—reaching 95% of the at-risk population in its study area. Among those tested, a small fraction showed evidence of infection, and more than 800 patients have already been treated with the single-dose therapy.
“Three pills, and then you’re treated,” said Elena Nicco, a principal investigator on the project. The simplicity of the regimen, she added, is key to reaching patients in remote settings where traditional care models fall short.
The implications extend beyond a single disease. Sleeping sickness has long been emblematic of the challenges facing neglected tropical diseases—conditions that disproportionately affect the poorest communities and attract limited commercial investment. Acoziborole offers a new model, showing how sustained public–private partnerships and targeted funding can deliver breakthroughs where they are most needed.
The World Health Organization(WHO) has set a goal of interrupting transmission of sleeping sickness by 2030. With a tool as simple as a single-dose cure, that ambition now appears within reach. But success will depend not only on scientific advances, but on continued investment in screening, surveillance, and health systems capable of delivering treatment at scale.
