In its late stage, Sleeping Sickness parasite crosses into the brain, causing confusion, seizures, severe sleep disturbance and, ultimately, death.
A new oral medicine could transform the fight against sleeping sickness, offering the first single-dose cure for both early and advanced stages of the disease.
The therapy, acoziborole, has received a positive scientific opinion from the European Medicines Agency for the treatment of gambiense human African trypanosomiasis in adults and adolescents weighing at least 40 kilograms.
If approved by regulators in endemic countries, it would replace treatments that require hospital stays, toxic injections, or ten days of carefully supervised medication.
Developed through a partnership between the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, the medicine is taken as a single oral dose of three tablets. The recommendation followed a pivotal Phase II/III clinical trial conducted in the Democratic Republic of Congo (DRC) and Guinea, in collaboration with national sleeping sickness control programmes.
For communities where sleeping sickness lingers, this breakthrough is profound
According to results published in The Lancet Infectious Diseases, the study demonstrated success rates of up to 96 per cent at 18 months across both early and advanced stages of the disease, with a favourable safety profile.
Dr Luis Pizarro, Executive Director of DNDi, said the progress was testament to the transformative power of collaborative science. “In just 20 years, we have gone from complicated treatments including arsenic derivatives with serious side effects, to today, when a single-dose, one-day therapy could safely cure patients,” he said.
For communities in remote parts of Central and West Africa, where sleeping sickness still lingers, the implications of this breakthrough are profound. Human African trypanosomiasis, or sleeping sickness, is transmitted by the bite of an infected tsetse fly and is almost always fatal without treatment.
Early symptoms include headaches and fever, but once the parasite crosses into the brain, it causes confusion, seizures, severe sleep disturbance and, ultimately, death.
The DRC carries the highest global burden of the disease, accounting for more than 80 per cent of reported cases over the past decade, according to DNDi. It is the only country that still records more than 1,000 new infections annually.
Kenya eliminated sleeping sickness as a public health problem last year
Yet even as the DRC grapples with the largest share of cases, other African nations are reaching important milestones.
In August last year, the World Health Organisation (WHO) validated Kenya as having eliminated sleeping sickness as a public health problem. Kenya becomes the tenth African country to achieve this status, having reported no indigenous cases since 2009. The last two cases, detected in 2012, were of infected tourists visiting the Masai Mara National Reserve.
For patients in isolated villages, existing treatments, including a 10-day course of oral medication introduced in 2018, can be nearly as difficult to access as injections. Travel costs, lost income and limited health infrastructure often interrupt treatment, risking relapse or continued transmission.
The development programme for acoziborole was rooted in African clinical research infrastructure. Trials were conducted in remote settings where the disease persists, led by local physicians and researchers.
Dr Erick Miaka, Director of the national sleeping sickness control programme in DRC, described it as a victory for Africa-led science, highlighting the role of doctors and researchers working in some of the continent’s most difficult-to-reach regions.
Producing treatments within Africa to strengthen supply resilience and local manufacturing capacity
Health leaders across Africa view the new treatment as more than a clinical advance. Africa CDC Director General Dr Jean Kaseya emphasised the importance of translating innovation into sustainable access, including exploring the possibility of producing treatments like acoziborole within Africa to strengthen supply resilience and local manufacturing capacity.
African Union Commissioner for Health, Humanitarian Affairs and Social Development, Ambassador Amma Adoma Twum-Amoah, added that the development represented a victory for health equity on the continent. “A shift from hospital beds into a simple cure, removing the last mile barriers to ending sleeping sickness for good,” she said.
Dr Michael Makanga of Global Health EDCTP3 noted that ongoing paediatric studies aim to turn the one-dose cure into a practical tool capable not only of treating individuals but of interrupting transmission.
The EMA’s Committee for Medicinal Products for Human Use granted its positive opinion through the EU-M4all pathway, a mechanism designed for high-priority medicines addressing unmet medical needs outside the European Union. The process engages World Health Organization experts and regulators from endemic countries, providing scientific endorsement that a medicine meets EU standards.
The opinion is expected to facilitate regulatory approvals in endemic countries, including the Democratic Republic of Congo, and may prompt an update to WHO treatment guidelines.
Sleeping sickness cases have declined by 98 per cent since 2001, when over 400,000 cases were reported
The WHO has set a target of eliminating Gambiense sleeping sickness as a public health problem by 2030. Cases have already declined by about 98 per cent since 2001, when nearly 40,000 cases were reported, and an estimated 300,000 went undiagnosed.
At that time, the only available treatment for late-stage disease was an injectable arsenic derivative with serious side effects.
More than two decades of investment in new therapeutic tools have yielded progressively improved treatments, including nifurtimox-eflornithine combination therapy in 2009 and the first oral treatment, fexinidazole, taken over ten days in 2018.
Today, fewer than 600 cases were recorded globally in 2024, according to data from DNDi and Sanofi.
The medicine will be provided free of charge to patients
Public-private partnerships have played a critical role in that decline. Sanofi has long supported sleeping sickness control efforts and announced that, once approved, acoziborole will be donated to WHO through its philanthropic organisation, Foundation S – The Sanofi Collective. The medicine will be provided free of charge to patients.
A separate study is underway to evaluate the drug’s use in children aged one to 14, expanding its potential reach.
While the results are promising, approval by regulators in endemic countries is still required. If approved and rolled out successfully, acoziborole could replace toxic and painful injections and bring the goal of eliminating sleeping sickness by 2030 closer to reality.
