Acoziborole, a new oral drug taken as a single three-pill dose, has won a positive opinion from a committee of the European Medicines Agency and could be available as soon as early next year. Public health experts say it could be a major step toward the World Health Organization’s goal of eliminating human African trypanosomiasis, or sleeping sickness, by 2030.
Sleeping sickness is transmitted by tsetse fly bites carrying the parasite Trypanosoma brucei gambiense, which causes more than 90% of current cases. The illness has an early stage of fever and headache and a later stage after the parasite crosses the blood-brain barrier, bringing neurological symptoms such as confusion, seizures and a disruption of sleep-wake cycles that gives the disease its name. Untreated, the infection can progress to coma and death.
Acoziborole treats both stages. It replaces older intravenous regimens that required clinic infrastructure, specialized staff and carried severe side effects, including painful vein reactions and, in some historic regimens, a roughly 5% treatment-related fatality rate. The current oral first-line medicine, fexinidazole, requires a 10-day course and can cause nausea, vomiting and heart-rhythm problems. Clinical trials of acoziborole reported only one notable side effect: mostly mild to moderate headaches.
Dr. Gerardo Priotto, who leads WHO’s program against sleeping sickness, emphasizes that past therapies were hard to deliver in the remote rural settings where most cases occur because they depended on equipment and reliable electricity and transport. Dr. Stéphane Hugonnet of the Drugs for Neglected Diseases Initiative, which developed acoziborole with Sanofi and partner funding, says the severe adverse effects of earlier drugs discouraged people from seeking care.
Because acoziborole is a single, well-tolerated dose, it could dramatically simplify treatment campaigns and expand access in isolated communities across savanna woodlands and lakeside vegetation where tsetse flies thrive. Sustained control measures — tsetse control, active case finding and treatment — have already reduced annual cases from hundreds of thousands in the past to about 1,000 today, with nearly two-thirds reported in the Democratic Republic of Congo (DRC). But the disease has historically ebbed and resurged, so easier, safer treatments could help prevent rebounds.
DNDi calls acoziborole transformative. Researchers at DNDi and partners ran trials in challenging settings such as South Ubangi province in the DRC, often building temporary facilities, training local staff, providing connectivity and arranging safe transport for participants. Much of the recent progress relies on African researchers and trial volunteers working under difficult conditions with limited infrastructure.
Ongoing research is testing whether rapid serologic blood tests could be used to start treatment immediately, rather than waiting for slower, more costly confirmatory examinations that are often unavailable in the field. Monica Mungier of Johns Hopkins cautions that the parasite can sometimes evade detection, so a negative test does not always exclude infection. Still, if simple blood tests prove reliable for guiding care, same-day testing and treatment would increase the number treated and shrink the human reservoir that sustains transmission.
Next steps include review by the DRC Ministry of Health and by WHO to determine whether treatment guidelines should be updated, which would help other countries authorize acoziborole. Global health specialists warn that reductions in Western funding could limit access to the drug in the places that need it most.
If acoziborole is rolled out alongside improved testing, sustained vector control and continued investment in surveillance and care, the single-dose therapy could markedly simplify delivery, extend treatment deeper into remote communities, and accelerate efforts to eliminate sleeping sickness.