KINSHASA / PARIS / GENEVA / AMSTERDAM – February 27, 2026
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Acoziborole Winthrop (acoziborole) as a single-dose oral treatment for both early and advanced-stage gambiense human African trypanosomiasis (sleeping sickness) in adults and adolescents aged 12 years and older weighing at least 40 kilograms. The therapy, co-developed by Drugs for Neglected Diseases initiative (DNDi) and Sanofi, demonstrated up to 96% success rates at 18 months in a pivotal Phase 2/3 study, marking a potential breakthrough in efforts to eliminate the disease by 2030.
Single-Dose Innovation for a Deadly Neglected Disease
The CHMP positive opinion was granted under an accelerated assessment procedure intended for high-priority medicines targeting unmet needs in countries outside the European Union. The recommendation is expected to facilitate approval in the Democratic Republic of Congo and support updates to World Health Organization (WHO) treatment guidelines, paving the way for broader access across Central and West Africa, where the disease remains endemic.
Sleeping sickness, transmitted by the tsetse fly, is almost always fatal if untreated. In its early stage, patients experience fever and headaches; in later stages, the parasite invades the central nervous system, causing neurological symptoms, behavioral changes, seizures, sleep disturbances, and ultimately death. Historically, treatment options were complex and often required prolonged hospitalization, including injectable arsenic derivatives with significant toxicity.
Acoziborole represents the first oral single-dose new chemical entity for this disease, offering a simplified regimen of three tablets administered in one day. Existing therapies require either a 10-day oral course or a combination of injections and oral medicines for advanced cases. The streamlined dosing approach could significantly reduce logistical burdens on fragile healthcare systems and expand access in remote settings.
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Clinical Evidence and Regulatory Pathway
The CHMP opinion is based on clinical and non-clinical data, including results from a Phase 2/3 study conducted in the Democratic Republic of Congo and Guinea in partnership with national sleeping sickness control programs. The study demonstrated high efficacy across both early and advanced stages, with a favorable safety profile and sustained cure rates up to 18 months.
Over the past two decades, sustained collaboration between DNDi, Sanofi, WHO, and endemic country partners has dramatically reduced reported cases of gambiense sleeping sickness—from nearly 40,000 in 1998 to fewer than 600 in 2024. Acoziborole builds upon previous therapeutic milestones, including nifurtimox-eflornithine combination therapy (NECT) and the first all-oral 10-day treatment, fexinidazole.
Sanofi has committed to donating Acoziborole Winthrop to WHO through Foundation S, ensuring the medicine will be available free of charge to patients in endemic regions. An additional clinical study is underway to evaluate the therapy in children aged one to 14 years.
Toward Elimination by 2030
The WHO has set a goal to eliminate gambiense sleeping sickness as a public health problem by 2030. The availability of a single-dose, field-friendly oral treatment could be transformative in accelerating case management, simplifying surveillance programs, and preventing disease resurgence in high-risk communities. By combining scientific innovation, regulatory acceleration, and philanthropic access commitments, Acoziborole may become a critical tool in global neglected disease control.
If approved in endemic countries, Acoziborole would represent a landmark achievement in neglected tropical disease drug development, demonstrating how public-private collaboration can deliver life-saving therapies for underserved populations.
Source: Sanofi press release
